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.”

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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