In­side Ver­tex 3.0: Can Resh­ma Ke­wal­ra­mani re­peat one of biotech's biggest suc­cess sto­ries 'a­gain and again and again'?

This was not how Reshma Kewalramani imagined spending her first day as Vertex CEO. The 47-year-old nephrologist should’ve been in a spacious window office on the 14th floor of the biotech’s glassy Boston Seaport headquarters, three rooms down from where she had spent the last three years. There should have been family photos on the desk, scientists buzzing in the labs beneath, and, feet away, executives she knew and trusted, briefing her on potential cures for sickle cell disease and diabetes.

Instead, on that bone-chillingly cold day last spring, she was at a makeshift desk in the dimly lit basement of her home outside Boston, a bivouac chosen because it was closest to the Wifi router. Her closest companion was Ferris Bueller’s smug face on the wall and she spent the day jumping from Zoom call to Zoom call, worried less about making new drugs than making sure her employees were safe and that the global supply chain didn’t leave a cystic fibrosis patient without access to the Vertex pills that had changed their life. It was April 1, 2020.

“These are not what I thought would be the two of the highest priorities,” she tells me. “The safety of our people? You take it for granted.”

The pandemic hit Vertex at the worst possible time. Over the last decade, CEO Jeffrey Leiden, a jovial but shrewd and commanding figure, had pushed the development of those CF drugs, turning the most common fatal genetic disease in US and Europe into, for 90% of patients, a treatable condition. In the process, they had gone from a $6 billion to a $60 billion company and won the rare collective awe of the business, medical and patient communities. “I scream it from the rooftops,” says Bob Coughlin, former CEO of industry group MassBio, whose 19-year-old son has CF. “He’s a whole new person, I’m filled with more gratitude than I’ve ever had in my whole damn life.”

Now, just as Leiden passed the torch, the entire world was collapsing. It was a trial by wildfire for Kewalramani, who had already been an unlikely choice as CEO. The heads of large biotechs are almost exclusively businesspeople, executives whose chief job is to sell the drugs the company has already developed and find other companies to acquire. If they have MDs, they also have an MBA or 20 years of experience in sales. All, historically, have been men.

Kewalramani was a clear-eyed, affable physician who had trained at Boston’s most prestigious hospitals and spent 12 years running trials at Amgen, but she had little experience on the business side of biotech. For the prior three years, leading Vertex’s medical team, she stood opposite the executive committee at key moments, explaining results from trials she designed and ran in sickle cell and cystic fibrosis.

She came from the medical side, which was unique,” says Terry McGuire, founder and general partner of the Boston-based biotech VC Polaris Partners. “It speaks to their desire to really focus on what’s going on in the clinic and for patients.” Indeed, Vertex had only considered physician-scientists for the role. They had big plans for the role — for what they called Vertex 3.0. Although they had become known as the CF company, for years, Leiden told anyone who would listen that he didn’t just want to transform one disease: He planned to use the lavish proceeds from those pills to cure CF completely and either cure or defang an Infernal Council of famous ailments: Sickle cell disease, diabetes, muscular dystrophy and pain, among others.

It was as ambitious a plan as a biotech had ever put forward, spanning medical disciplines from hematology to nephrology and technologies from old-fashioned pills to new forms of CRISPR gene editing, and they needed someone with unimpeachable scientific chops to carry it out. If Kewalramani and her team can, they will change the face of medicine: Not just for one rare disease but several, and a few not so rare ones as well. They could also set off the same string of rancorous global debates that have followed Vertex’s CF drugs, as the company charged more than what many countries said they could pay. Kewalramani, while striking a less abrasive tone than her predecessor, has pledged to keep the same pricing strategy moving forward.

“We’re going to do what we did in CF,” Kewalramani tells me, echoing a promise she makes repeatedly. “Again and again and again.” But first they would have to deal with Covid-19.

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Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Tony Johnson, Goldfinch Bio CEO (Goldfinch via YouTube)

Kid­ney dis­ease drug­mak­er Goldfinch Bio shuts down

Goldfinch Bio, attempting to make treatments for kidney diseases and diabetic nephropathy, is shutting down.

President and CEO Tony Johnson confirmed to Endpoints News Friday afternoon that the biotech shut down after “fundraising challenges in the current macro-environment.” Fierce Biotech first reported the news.

Johnson, who joined in 2017 after a stint as SVP of early clinical development at AstraZeneca, said in a text that the company “entered the ABC process recently,” referring to an assignment for the benefit of the creditors, which provides a different wind-down avenue than a bankruptcy.

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Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Dutch biotech starts liq­ui­da­tion af­ter end­ing PhI­II in GVHD

A 13-year-old Dutch biotech is going through a liquidation process after an unexpected end to its Phase III trial testing whether its combination of two monoclonal antibodies was superior to Incyte’s Jakafi.

Xenikos had hoped to prove its investigational therapy, named T-Guard, was better than Jakafi at garnering a complete response in patients experiencing life-threatening complications in which new cells from a hematopoietic stem cell transplant begin to fight the body. Jakafi was approved for the indication, steroid-refractory acute graft-versus-host disease, in May 2019.

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#JPM23: BeiGene CEO John Oyler on the com­pa­ny's big am­bi­tions

BeiGene CEO John Oyler and Endpoints News Executive Editor Drew Armstrong spoke one-on-one about where the drugmaker is headed in its quest to become a diversified multinational, and what it will take to get there. This transcript has been edited for brevity and clarity.

Drew Armstrong:

I am joined today by BeiGene CEO John Oyler. By way of introduction, John and I met about a little over three years ago for the first time in Beijing, when I was doing some reporting. I got to travel around the country and I had this huge notebook full of reporting on interesting things that his company was doing and some of the other companies were doing over there. And I came back to the states ready to write a great magazine story about BeiGene and about innovation that was happening globally. And then I think we all know what happened about a week and a half after I returned, in January of 2020. And unfortunately that work never saw the light of day.

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Robert Hariri, Celularity CEO (Vivien Killilea/Getty Images for Brain Mapping Foundation)

Celu­lar­i­ty warns of loom­ing lay­offs, spot­lights ear­ly pro­grams as mon­ey runs dry

Celularity has warned “a substantial portion” of its employees that layoffs could be looming in the face of a reprioritization effort. In the same breath, it touted some of its very early next-gen candidates in degenerative diseases and oncology.

“While Celularity is hopeful that there will be no need to substantially reduce its headcount, the notifications were provided in accordance with applicable law and to maintain full transparency with its employees,” the news release reads, in part.