Left to right: Arthur Pappas, Robert Nelsen, Peter Kolchinsky Doug Cole and David Beier

In rare po­lit­i­cal for­ay, top biotech in­vestors urge Con­gress to re­ject drug pric­ing bill

Thir­teen of the top biotech ven­ture cap­i­tal­ists in the coun­try wrote a let­ter last week warn­ing law­mak­ers that if Con­gress pass­es a drug pric­ing bill House Speak­er Nan­cy Pelosi has put be­fore law­mak­ers, they won’t be able to in­vest in bio­med­ical re­search at their cur­rent rate, and pa­tients will suf­fer.

“If poli­cies such as those in­clud­ed with­in H.R. 3, the Low­er Drug Costs Now Act, are passed, our abil­i­ty to con­tin­ue to in­vest in fu­ture bio­med­ical in­no­va­tion will be se­vere­ly con­strained, thus crush­ing the hopes of mil­lions of pa­tient wait­ing for the next break­throughs to treat or cure their can­cers, rare ge­net­ic dis­eases, Alzheimer’s, or oth­er se­ri­ous and life-threat­en­ing con­di­tions,” they wrote in a let­ter ad­dressed to the high­est-rank­ing De­moc­rats and Re­pub­li­cans in the House and Sen­ate and ac­quired by End­points News. 

The list of sig­na­to­ries in­cludes Arch Ven­ture Part­ners’ Robert Nelsen, Bay City Cap­i­tal’s David Beier, Flag­ship Pi­o­neer­ing’s Doug Cole, RA Cap­i­tal Man­age­ment’s Pe­ter Kolchin­sky and Pap­pas Cap­i­tal’s Arthur Pap­pas. They write they have over $20 bil­lion in­vest­ed in pri­vate and pub­lic bio­phar­ma­ceu­ti­cal com­pa­nies.

The in­vestors join the larg­er phar­ma com­pa­nies in ex­press­ing alarm about the price con­trols in Pelosi’s bill. Pfiz­er is urg­ing vot­ers to “Tell Con­gress: Re­ject the Dan­ger­ous Drug Pric­ing Bill.”

While the large phar­ma com­pa­nies have long ped­dled in Wash­ing­ton through the lob­by­ing group PhRMA and have amped spend­ing to record lev­els as drug pric­ing re­form has gained trac­tion, biotech ven­ture cap­i­tal­ists have large­ly been more re­luc­tant to en­ter the po­lit­i­cal fray. In­creas­ing­ly, though, the de­bate around drug pric­ing re­form and what it might mean for R&D has cen­tered around the small­er biotechs these VC funds keep afloat.

Bob­by Dubois NPC

Last week, STAT ran a piece quot­ing tiny biotechs as fear­ing a “nu­clear win­ter” if the Nan­cy Pelosi-backed bill passed. In Oc­to­ber, Bob­by Dubois, the CSO of the in­dus­try-backed Na­tion­al Phar­ma­ceu­ti­cal Coun­cil told End­points that he couldn’t be sure that a fall in rev­enue for big phar­ma would lead to cuts to R&D spend­ing but that it was very like­ly that such a bill would cut off dol­lars from small­er biotechs.

Biotechs’ high start­up costs and at­tri­tion rate, the the­o­ry goes, make them de­pen­dent on huge po­ten­tial pay­outs. With­out those, in­vest­ments would dry up.

The bill would al­so like­ly de­rail the biotech IPO mar­ket, which is how ven­ture cap­i­tal­ists have earned much of their mon­ey in bio­phar­ma in re­cent years.

Un­veiled in Sep­tem­ber, the De­mo­c­ra­t­ic bill — which con­tains el­e­ments Re­pub­li­cans have backed but has lit­tle chance of pass­ing the Sen­ate — would teth­er the price of the na­tion’s 250 most ex­pen­sive drugs to an in­ter­na­tion­al price in­dex and all-but-force com­pa­nies to ne­go­ti­ate the fi­nal fig­ure with the fed­er­al gov­ern­ment. An ear­ly Con­gres­sion­al Bud­get Of­fice analy­sis es­ti­mat­ed the bill would re­duce fed­er­al spend­ing on Medicare Part D by $345 bil­lion be­tween 2023 and 2029 but al­so lead to be­tween 8 and 15 few­er drugs be­ing brought to mar­ket over the next 10 years, al­though the CBO said the ef­fect on in­no­va­tion was dif­fi­cult to quan­ti­fy.

Dar­ren So­to

In the let­ter, the VCs ar­gued such a sys­tem would be “grant­i­ng the U.S. gov­ern­ment es­sen­tial­ly unchecked au­thor­i­ty” to set prices and pin those prices to coun­tries that “sys­tem­at­i­cal­ly un­der­val­ue” med­ical break­throughs.

“Biotech in­vest­ment al­ready is a high-risk en­ter­prise…” they wrote. “If, af­ter we in­vest hun­dreds of mil­lions of dol­lars over a decade or more to achieve such suc­cess, the gov­ern­ment can im­pose an ar­ti­fi­cial­ly low price on the few new drugs that make it to mar­ket, our ro­bust biotech in­vest­ment ecosys­tem will be­come un­sus­tain­able.”

The dis­agree­ment be­tween the VCs and De­mo­c­ra­t­ic and some Re­pub­li­can law­mak­ers is both around whether the cuts will af­fect in­no­va­tion and the val­ue of some of that in­no­va­tion. The VCs de­scribed the ther­a­pies they back as “mir­a­cle drugs, with the abil­i­ty not just to treat, but to ac­tu­al­ly cure, so many ge­net­i­cal­ly-based dis­eases and oth­er se­ri­ous con­di­tions.”

An­na Es­hoo

In past push­es for drug pric­ing re­form, ad­vo­cates have most­ly said that that rhetoric is over­stat­ed and pric­ing con­trols will leave R&D spend­ing most­ly un­touched. But a few De­moc­rats, in­clud­ing Flori­da Rep­re­sen­ta­tive Dar­ren So­to and Cal­i­for­nia Rep­re­sen­ta­tive An­na Es­hoo, now ar­gue an uptick in ac­cess is worth los­ing a few drugs.

“Three hun­dred forty-five bil­lion dol­lars in sav­ings ver­sus the cost of eight to 15 few­er drugs over 10 years,” So­to said at a re­cent hear­ing be­fore the House En­er­gy and Com­merce Com­mit­tee, ac­cord­ing to STAT. “I frankly think it’s worth it.”

Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Paul Hudson, Getty Images

Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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FDA ex­pert pan­el unan­i­mous­ly rec­om­mends ap­proval for Hori­zon Ther­a­peu­tics eye drug

An FDA advisory committee noted with concern a small safety database but unanimously endorsed a Horizon Therapeutics drug for a rare eye autoimmune disease that can blind patients: teprotumumab for thyroid eye disease (TED).

“It was a pretty easy vote,” said Erica Brittain, an NIH biostatistician and one of the 12 panelists on FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee.

This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

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On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.