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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2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Eisai and METAvivor plan to debut the latest 'This is MBC' campaign at the San Antonio Breast Cancer Symposium (SABCS).

Ei­sai re-ups metasta­t­ic breast can­cer aware­ness cam­paign with strik­ing pa­tient pho­tographs

Eisai is debuting the newest ads in its long-running “This is MBC” campaign this week. In what’s become an annual tradition, Eisai and metastatic breast cancer advocacy partner METAvivor will show the striking photographs of people living with metastatic breast cancer first at the San Antonio Breast Cancer Symposium (SABCS).

The new “Imagine” campaign features 12 patients photographed around waterfalls to symbolize that same kind of sudden drop into a pool that MBC causes in a person’s life, said Beth Fairchild, co-founder of #CancerCulture who was the president of METAvivor six years ago when the campaign began. Fairchild, who is living with MBC, has helped create all of the annual “This is MBC” campaigns.

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Pfiz­er and BioN­Tech look to toss Mod­er­na patent suit, call­ing claims 'unen­force­able'

Pfizer and BioNTech took a swing at Moderna’s Covid-19 patent claims in Massachusetts federal court on Monday, calling them “invalid,” “overbroad” and “unenforceable.”

The defendants also filed counterclaims against the Cambridge, MA-based biotech, seeking a dismissal of the case, recovery of court fees and an official judgment invalidating Moderna’s claims.

Moderna sued Pfizer and BioNTech back in August, alleging that the partners’ Covid-19 vaccine Comirnaty copied parts of Moderna’s vaccine technology patented before the pandemic, when it was developing an mRNA vaccine for MERS, another respiratory illness.

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Glen­mark hit with warn­ing let­ter over pro­ce­dures, qual­i­ty con­trol is­sues at In­dia man­u­fac­tur­ing plant

The generics producer Glenmark Pharmaceuticals has been handed a warning letter by US regulators.

The letter, which was sent to the manufacturer on Nov. 22, noted issues from an inspection over the summer at Glenmark’s facility in the town of Colvale, India, in the state of Goa.

According to the letter, the FDA found that Glenmark’s investigation of rejected batches of drugs “failed to extend to other batches, dosage strengths, and drug products.” The warning letter also noted that the site had failed to establish “adequate written procedures” for production and process control to ensure drugs have the correct strength, quality and purity.

Klick Health is lighting the way, literally, this holiday season to encourage connection for lonely seniors in long-term care facilities.

Klick Health an­nu­al hol­i­day spot­light se­nior lone­li­ness and the pow­er of con­nec­tion

Every year Klick Health leans into a cause for the holidays, and this year it’s highlighting the sometimes lonely season for seniors. So Klicksters, as employees call themselves, decided to brighten one nursing home community in hopes of inspiring others to do the same.

Klick literally lit up the Tony Stacey Centre for Veterans Care, a long-term care home in Toronto where 75% of residents receive no visitors during the holiday season. The agency brought staff and family along with lighting crews and musicians for a “Light the Way” event, creating a video of the experience debuting on Tuesday.

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Sum­i­to­vant sub­sidiaries En­zy­vant and Al­ta­vant merge in­to com­bined com­pa­ny

Two Sumitovant Biopharma entities are merging under one name, effective immediately.

Enzyvant Therapeutics and Altavant Sciences announced they have merged to form a singular entity focused on developing therapies for patients with rare diseases. The combined company will keep the name Enzyvant and along with clinical development will eventually include in-house manufacturing.

Bill Symonds, the current CEO of both Altavant and Enzyvant, is now CEO of the merged company.

Eu­ro­pean Com­mis­sion lays ground­work to un­wind Il­lu­mi­na's $7B+ Grail merg­er

The European Commission has recommended steps that — though not yet final — would require Illumina to “swiftly” unwind its controversial $7.1 billion Grail buyout.

The Commission delivered a “statement of objections” on Monday, detailing the process Illumina would need to take in divesting Grail, its blood testing spinout launched in 2016. Illumina re-acquired Grail back in August, despite criticism from both the FTC and EU.