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.”

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

The drug, known as ofatumumab or Arzerra, has performed consistently well across late-stage trials in patients with the most common form of MS, including in head-to-head studies against Sanofi’s old blockbuster Aubagio. But, if the drug is approved, Novartis will find itself in a crosstown game of catch-up; since a 2017 approval, Roche’s Ocrevus has become the second best-selling MS drug on the market, nearly eclipsing Biogen’s Tecfidera last quarter with over a $1 billion in sales.

Coro­n­avirus out­break threat­ens short­age of 150 drugs — re­port

American patients who suffer from conditions other than Covid-19 could feel the impact of the coronavirus due to shortage of drugs — as 150 prescription drugs are now reportedly on a list of at-risk therapies. The list spans “antibiotics, generics and some branded drugs without alternatives,” Axios reported citing sources familiar with the list. The FDA declined to comment.

Although factories in China are gradually reopening, restrictions in travel and disruptions at transit hubs are still slowing down production. An Indian company that relies on active pharmaceutical ingredients (API) from China told Bloomberg last week that it’s seeing prices of commonly used drugs jump by 40% to 70%.

Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.

Tim Mayleben (file photo)

Es­pe­ri­on's goldilocks cho­les­terol fight­er wins FDA ap­proval — will its 'tra­di­tion­al' pric­ing ap­proach spur adop­tion?

It’s more effective than decades-old statins but not as good as the injectable PCSK9 — the goldilocks treatment for cholesterol-lowering, bempedoic acid, has secured FDA approval.

Its maker, Esperion Therapeutics, is betting that their pricing strategy — a planned list price of between $10 to $11 a day — will help it skirt the pushback the PCSK9 cholesterol fighters, Repatha and Praluent, got from payers for their high sticker prices.

The sky-high expectations for the pair of PCSK9 drugs that were first approved in 2015 quickly simmered — and despite a 60% price cut, coupled with data showing the therapies also significantly cut cardiovascular risk, sales have not really perked up.

Esperion is convinced that by virtue of being a cheaper oral therapy, bempedoic acid will hit that sweet spot in terms of adoption.

“We’re kind of like the old comfortable shoe,” Esperion’s chief commercial officer Mark Glickman remarked in an interview with Endpoints News ahead of the decision date. “It’s an oral product, once-daily and nontitratable — these are things that just resonate so true with patients and physicians and I think we’ve kind of forgotten about that.”

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Drug ap­provals: FDA pub­lish­es dataset of CDER ap­provals since 1985

To provide researchers with more accurate and accessible data about historic drug approvals, the FDA on Friday released a dataset containing information about all new drugs and biologics approved by the Center for Drug Evaluation and Research (CDER) dating back to 1985.

“FDA created the compilation to facilitate data accessibility, transparency, and accuracy when researchers seek information about an approved drug,” the FDA writes, adding that the compilation should accurately reflect “the state of each application at the time of initial regulatory approval.”

Deborah Dunsire

The fourth CGRP mi­graine drug is here. Time for Lund­beck to prove it's worth $2B

They may be late, but Lundbeck is now officially in the game for preventing migraine with CGRP drugs.

The FDA has OK’d eptinezumab, the prize in Lundbeck’s $2 billion acquisition of Alder. Like rival offerings from Amgen/Novartis, Eli Lilly and Teva, the antibody blocks the calcitonin gene-related peptide, which is believed to dilate blood vessels in the brain and cause pain.

It will now be sold as Vyepti. The company has yet to announce a price. Amgen and Novartis had set the wholesale acquisition cost of their pioneering Aimovig at $6,900 for a year’s supply before raising it slightly this year; Lilly and Teva had followed suit with Emgality and Ajovy.

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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