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Vertex Pharmaceuticals

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Vertex Pharmaceuticals Incorporated
Company typePublic
Industry
Founded1989; 35 years ago (1989)
HeadquartersBoston, Massachusetts, U.S.
Key people
ProductsPharmaceuticals
RevenueIncrease US$9.87 billion (2023)
Decrease US$3.83 billion (2023)
Increase US$3.62 billion (2023)
Total assetsIncrease US$22.7 billion (2023)
Total equityIncrease US$17.6 billion (2023)
Number of employees
c. 5,400 (2023)
Websitevrtx.com
Footnotes / references
[1][2]

Vertex Pharmaceuticals Incorporated is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headquarters in Boston, Massachusetts, and three research facilities, in San Diego, California, and Milton Park, Oxfordshire, England.

History

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Vertex was founded in 1989 by Joshua Boger[3] and Kevin J. Kinsella[4] to "transform the way serious diseases are treated."[5]

The company's beginnings were profiled by Barry Werth in the 1994 book The Billion-Dollar Molecule.[4] His 2014 book, The Antidote: Inside the World of New Pharma, chronicled the company's subsequent development over the next two decades.[6]

By 2004, its product pipeline focused on viral infections, inflammatory and autoimmune disorders, and cancer.[7]

In 2009, the company had about 1,800 employees, including 1,200 in the Boston area.[3] By 2019 there were about 2,500 employees.[8]

Since late 2011, Vertex has ranked among the top 15 best-performing companies on the Standard & Poor's 500. Vertex shares increased 250 percent in the same period.[9] In January 2014, Vertex completed its move from Cambridge, Massachusetts, to Boston, Massachusetts, and took residence in a new, $800 million complex. Located on the South Boston waterfront, it marked the first time in the company's history that all of the roughly 1,200 Vertex employees in the Greater Boston area worked together.[10]

On 23 January 2019, Ian Smith, the COO and interim CFO of Vertex, was terminated from his position for undisclosed personal behavior that violated established company code of conduct rules.[11] In June of the same year, Vertex announced it would acquire Exonics Therapeutics for up to $1 billion and collaborate with CRISPR Therapeutics, boosting its development of treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1.[12][13]

In September 2019 the company announced it would acquire Semma Therapeutics for $950 million in cash.[14] Semma Therapeutics created a "small, implantable device that holds millions of replacement beta cells, letting glucose and insulin through but keeping immune cells out."[15]

On 1 April 2020, Reshma Kewalramani, then the chief medical officer, became president and chief executive officer of Vertex Pharmaceuticals.[16] Former CEO and president Jeffrey Leiden transitioned to the role of executive chairman of the board of directors, as of 1 April 2020.[16] Leiden was to serve as executive chairman until April 2023,[17][18] although As of September 2024 he remains in that post.[19]

In July 2022, the business announced it would acquire ViaCyte, Inc. for $320 million.[20]

In April 2024, it was announced Vertex had agreed to acquire the Seattle-headquartered clinical stage biopharmaceutical company Alpine Immune Sciences for $4.9 billion.[21]

Medicines

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Ivacaftor, lumacaftor and tezacaftor

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In 2012 ivacaftor was designated as an orphan drug, identifying cystic fibrosis as affecting fewer than 200,000 people in the United States. On 31 January 2012, Vertex gained FDA approval[22] of the first drug, Kalydeco,[23] to treat the underlying cause of cystic fibrosis rather than the symptoms, in patients 6 years or older who have the G551D gene mutation.

In the US, 30,000 people have cystic fibrosis. About 4% of those, or 1,200, have the G551D gene mutation. In 2017 Vertex marketed the drug for $311,000 per person per year.[24][25]

Vertex also studied ivacaftor in combination with another drug (lumacaftor[26]) for the most common mutation in cystic fibrosis (CF), known as F508del, and published the first set of results in 2012. Vertex produced the drug after 13 years of research and development, with $70 million in support from the Cystic Fibrosis Foundation.[27][28]

In the UK, the company provided the drug free for a limited time for certain patients. Subsequently, the hospitals decided to continue to pay for the drug for those patients. UK agencies estimated the cost per quality adjusted life year (QALY) at between £335,000 and £1,274,000—far above the NICE thresholds[29] of £20,000-£30,000.

On 5 November 2014 Vertex announced the submission of a New Drug Application (NDA) to the FDA for a fully co-formulated combination of lumacaftor and ivacaftor for people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.[30] In 2015, FDA approved the combination of lumacaftor and ivacaftor to treat CF in patients 12 years and older, who have the F508del mutation. The combination drug is marketed under the tradename Orkambi.[31]

On 28 March 2017, Vertex announced Phase 3 data from a dual combination treatment, tezacaftor plus ivacaftor, in patients with cystic fibrosis.[32] On 12 February 2018, the FDA approved the combination, marketed as Symdeko.[33]

Triple combination treatments

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In 2016, Vertex began developing a new group of CFTR modulators in combination with tezacaftor and ivacaftor.[33] In 2017, the company reported results that showed benefits for patients with different mutations that represent 90% of the CF population.[34] On 22 October 2019, two months after a new drug application was filed with it, the FDA approved Vertex's Triple-combo therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) for patients 12 and older with at least one F508del mutation.[35] The FDA approval covers approximately 90% of patients with cystic fibrosis.[36]

Vertex has refused to make Trikafta available in developing countries and works to block generic alternatives, making it inaccessible for thousands of patients.[37]

Genetic therapies

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Historical

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Telaprevir

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Main article: Telaprevir

In 2014, Vertex discontinued telaprevir.[44] In May 2011, the Food and Drug Administration (FDA) had approved the drug telaprevir (Incivek) as an oral treatment for hepatitis C. Development and commercialization of telaprevir was shared with Johnson & Johnson for European distribution and Mitsubishi for Asia. Telaprevir is a protease inhibitor.[45]

CEO compensation

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In 2015, several media outlets reported CEO Jeffrey Leiden's 2014 compensation to be approximately US$48.5 million.[46] Vertex shareholders opted for a reduction in CEO compensation in 2015 and 2016, resulting in 2016 earnings of US$17.4 million.[47]

Pricing in the UK

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The initial pricing of Orkambi at US$135,000 to US$270,000 per patient per year, depending on the patient's country, led to campaigns by parent groups concerned that it limited access to the drug for children and young adults with cystic fibrosis, and also led to allegations of unfair pricing by the UK's National Health Service.[48][49][50][51][52]

In March 2019, Vertex was legally required to destroy 7,880 packs of Orkambi that reached their expiry dates during price negotiations with the NHS.[53][54]

On October 24, 2019, NHS England agreed to fund wider access to all of the pharmaceutical company's cystic fibrosis medications that were already licensed at that time, such as Orkambi, Symkevi and Kalydeco, and any future indications of these medicines. The funding agreement was finalized soon after Vertex signed reimbursement deals elsewhere, including NHS Scotland, Spain and Australia.[55][36] Vertex agreed to offer equivalent pricing to the NHS in Wales and Northern Ireland .[56] On June 30, 2020, Vertex and NHS expanded the agreement to include reimbursement of Kaftrio (the name used for marketing Trikafta in the UK), making England one of the first countries in Europe to fund it.[57]

NICE in October 2023 declined to recommend Kaftrio, Symkevi and Orkambi for use in CF patients, saying they were too expensive to be covered. “The most likely cost-effectiveness estimates for [Kaftrio, Orkambi and Symkevi] are above the range that NICE considers an acceptable use of NHS resources,” the agency wrote. Acceptable pricing terms were finally agreed in June 2024.[58]

Global pricing

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Pricing and public funding varies by country beyond the USA and the UK.

By October 2019, Vertex's CF medicines were publicly funded in 17 countries.[59]

By 2023, Vertex had reimbursement agreements in 36 countries for their CF medicines, of which 35 are considered high-income countries. About 27% (51,322) of the estimated CF patients were treated with Trikafta, of whom nearly half (49%) lived in the U.S., 42% in Europe, and 9% in Canada, Israel, Australia, and New Zealand combined.[60]

As of 2024, a large proportion of diagnosed patients in South America, Asia, and Africa had no subsidies for Trikafta, and the therapy was inaccessible in Mexico and Cuba.[60] Some families found that it was cheaper to fly to Argentina to purchase a generic version there.[61]

Researchers stated that prices sought by Vertex were “prohibitively high”, and "likely to result in a profound international divergence in outcomes.” They suggested possible solutions including price reductions under voluntary licensing agreements, challenging the patent conditions, or compulsory license applications, often provided by governments to allow another company to produce the medication without the consent of the patent holder.[60]

Venture philanthropy

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In the late 1990s the Bethesda-based Cystic Fibrosis Foundation, encouraged by then-President Robert Beall, began investing in Vertex— when it was a small start-up biotechnology company— to help fund the development of Kalydeco in the form of venture philanthropy. The total investment amounted to $150 million.[62] In 2014, the CF Foundation sold the rights to the royalties of the drugs for $3.3 billion, twenty times the foundation's 2013 budget.[62][63] Proponents of venture philanthropy say the high financial return helps speed drug development and also provides potential monetary rewards that can go to more research.[62]

By 2015 the annual price of Kalydeco had been increased to more than $300,000 per patient.[64]

According to an article published in the Milwaukee Journal Sentinel Vertex executives "grossed more than $100 million by cashing in stocks and stock options" and at "one point, the value of company's stock increased more $6 billion in a single day."[65]

Twenty-nine physicians and scientists working with people with cystic fibrosis (CF) wrote to Jeff Leiden, CEO of Vertex Pharmaceuticals to plead for lower prices.[66]

We are aware of the financial complexities of the huge expenses for R & D with respect to the small number of patients or the market system that enables these advances to become reality. Yet – notwithstanding all your patient support programs – it is at best unseemly for Vertex to charge our patients' insurance plans (including strapped state medical assistance plans), $294,000 annually for two pills a day (a 10-fold increase in a typical patient's total drug costs). This action could appear to be leveraging pain and suffering into huge financial gain for speculators, some of whom were your top executives who reportedly made millions of dollars in a single day (Boston Globe, 29 May).

— David M. Orenstein, MD et al.

The company responded in an email that "while publicly funded academic research provided important early understanding of the cause of cystic fibrosis, it took Vertex scientists 14 years of their own research, funded mostly by the company, before the drug won approval."[65]

On 15 April 2015 in Cambridge, Massachusetts, Joan Finnegan Brooks of the Cystic Fibrosis Foundation spoke about the role of Vertex and venture philanthropy to a panel of biotech leaders hosted by Life Sciences Foundation on the topic of patient advocacy in the biotech industry.[67] While Brooks, who has cystic fibrosis, expressed gratitude for Vertex's development of Kalydeco, she observed that "More than 25% of people are saying (in surveys conducted by the CFF) that they are skipping medications or delaying medications or skipping doctor appointments because of cost of care issues." She added that "one of the things that the Foundation has done," is to "develop resources that can help patients bridge that gap through patient access programs, and so forth."[68]

See also

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References

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  1. ^ "Vertex Pharmaceuticals Inc. 2023 Annual Report (Form 10-K)". U.S. Securities and Exchange Commission. 15 February 2024.
  2. ^ "Vertex Pharmaceuticals Incorporated". Hoover's. Retrieved 24 June 2012.
  3. ^ a b Weisman, Robert (24 April 2011). "Rearranging the Corporate DNA". Boston Sunday Globe. p. G1. Retrieved 25 June 2012.
  4. ^ a b Werth, Barry (1994). The Billion-Dollar Molecule: One Company's Quest for the Perfect Drug. New York: Simon & Schuster. ISBN 0671510576. OCLC 32047662. The Billion-Dollar Molecule at Google Books.
  5. ^ Roth, Michael S. (21 May 2011). "Why liberal arts matter". CNN. Retrieved 7 January 2022.
  6. ^ Werth, Barry (2014). The Antidote: Inside the World of New Pharma. New York: Simon & Schuster. ISBN 9781451655667. OCLC 859375019.
  7. ^ "Vertex Pharmaceuticals Reports Fourth Quarter and Full Year 2004 Financial Results and Full Year 2005 Guidance". Vertex Pharmaceuticals. Retrieved 30 May 2019.
  8. ^ "Vertex Pharmaceuticals on the Forbes Global 2000 List". Forbes. Retrieved 30 May 2019.
  9. ^ Robert Weisman (17 April 2015), Vertex chief's compensation totaled $36.6 million last year: Total included a retention bonus, Boston Globe, retrieved 19 July 2015
  10. ^ Weisman, Robert (4 February 2014). "New complex unites Vertex employees". The Boston Globe. Retrieved 30 May 2019.
  11. ^ Owens, Jeremy C. (23 January 2019). "Vertex terminates COO for 'personal behavior'". MarketWatch. Retrieved 25 January 2019.
  12. ^ "Vertex Expands into New Disease Areas and Enhances Gene Editing Capabilities Through Expanded Collaboration with CRISPR Therapeutics and Acquisition of Exonics Therapeutics". BioSpace.
  13. ^ "Vertex Dives into DMD with Exonics Acquisition and CRISPR Therapeutics Partnership Expansion". BioSpace. 7 June 2019.
  14. ^ "Vertex to buy cell therapy developer Semma for $950 million". 3 September 2019. Archived from the original on 9 July 2020 – via uk.reuters.com.
  15. ^ Time Staff. "12 Innovations That Will Change Health Care and Medicine in the 2020s". Time. Retrieved 29 October 2019.
  16. ^ a b DeAngelis, Allison (2 April 2020). "VERTEX 3.0: One of the area's largest biotechs enters new era of leadership, drug development". www.bizjournals.com. Retrieved 6 April 2020.
  17. ^ "Vertex's Reshma Kewalramani takes CEO post as longtime vet Jeff Leiden moves to new role". Endpoints News. Retrieved 31 July 2019.
  18. ^ "Vertex Pharmaceuticals' Jeffrey Leiden to step aside as CEO". STAT. 25 July 2019. Retrieved 31 July 2019.
  19. ^ "Jeffrey Leiden, M.D., Ph.D." Vertex.
  20. ^ "Vertex to Acquire ViaCyte, with the Goal of Accelerating its Potentially Curative VX-880 Programs in Type 1 Diabetes". 11 July 2022.
  21. ^ "Vertex to acquire Alpine Immune Sciences for $4.9bn - Pharmafile". pharmafile.com. 11 April 2024. Retrieved 11 April 2024.
  22. ^ "FDA approves Kalydeco to treat rare form of cystic fibrosis" (Press release). Food and Drug Administration. 31 January 2011. Retrieved 24 June 2012.
  23. ^ "KALYDECO™ (ivacaftor) tablets". Vertex Pharmaceuticals. Retrieved 24 June 2012.
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  25. ^ Herper, Matthew. "For Vertex Pharmaceuticals, Can One Billion-Dollar Breakthrough Beget Another?". Forbes. Retrieved 10 June 2019.
  26. ^ "VX-809 (Cystic Fibrosis)". Vertex Pharmaceuticals. 7 September 2011. Retrieved 24 June 2012.
  27. ^ "Vertex Wins Approval for Kalydeco to Treat Cystic Fibrosis". 31 January 2012. Retrieved 10 June 2019.
  28. ^ "Xconomy: Vertex Seeks FDA Green Light for Cystic Fibrosis Drug, Second Potential Hit of Big Year". Xconomy. 19 October 2011. Retrieved 10 June 2019.
  29. ^ Deborah Cohen; James Raftery (12 February 2014). "Orphan Drugs: Paying twice: questions over high cost of cystic fibrosis drug developed with charitable funding". BMJ. 348 (feb12 1): g1445. doi:10.1136/bmj.g1445. PMID 24523379. S2CID 8702814.
  30. ^ "Vertex Submits Applications in the U.S. and Europe for Approval of Lumacaftor in Combination with Ivacaftor for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation | Vertex Pharmaceuticals". investors.vrtx.com.
  31. ^ "Press Announcements – FDA approves new treatment for cystic fibrosis". www.fda.gov (Press release). Office of the Commissioner. Retrieved 16 January 2017.
  32. ^ "Is Vertex's New CF Drug Unprecedented? Incremental? Expensive? Maybe All Three". Forbes. 29 March 2017.
  33. ^ a b "Vertex and Treating CF: High Hopes for Triple Combinations and Gene Therapy Possibilities". Cystic Fibrosis News Today. 17 September 2018.
  34. ^ "'Just What We Dreamed.' New Vertex Drugs Show Dramatic Benefit Against Cystic Fibrosis". Forbes. 18 July 2017.
  35. ^ "FDA approves new breakthrough therapy for cystic fibrosis". FDA. 21 October 2019.
  36. ^ a b Parsons, Lucy (24 October 2019). "Vertex, NHS England and NICE finally reach agreement for Orkambi". PMLive. Retrieved 29 October 2019. The triple therapy, which has just received approval from the FDA in the US, is thought to be effective in up to 90% of CF patients.
  37. ^ Nolen, Stephanie; Robbins, Rebecca (7 February 2023). "The Drug Is a 'Miracle' but These Families Can't Get It - Vertex Pharmaceuticals is not making its drug, Trikafta, available in poorer countries, where thousands of diagnosed patients stand to benefit". The New York Times. ISSN 0362-4331. Retrieved 29 March 2023.
  38. ^ "Vertex, CRISPR Tx Team up on Gene Editing Drugs in $105M Pact". Exome. 26 October 2015.
  39. ^ CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001™ for the Treatment of Severe Hemoglobinopathies, PM Vertex May 11, 2020; retrieved May 11, 2020
  40. ^ "Vertex, Moderna Launch Cystic Fibrosis Collaboration". News: Discovery & Development. Genetic Engineering & Biotechnology News (Paper). 36 (14): 17. August 2016.
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  42. ^ DeAngelis, Allison (3 January 2019). "Vertex teams up with Feng Zhang startup on gene editing tech". www.bizjournals.com. Retrieved 10 June 2019.
  43. ^ "Pipeline | Vertex Pharmaceuticals". www.vrtx.com.
  44. ^ Silverman, Ed (12 August 2014). "From Riches to Rags: Vertex Discontinues Incivek as Sales Evaporate". Pharmalot (Blog). Dow Jones & Company. Retrieved 13 August 2014.
  45. ^ "Approval of Incivek (telaprevir), a direct acting antiviral drug (DAA) to treat hepatitis C (HCV)" (Press release). Food and Drug Administration. 24 May 2011. Retrieved 25 June 2012.
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  48. ^ Thomas, Katie (24 June 2018). "A Drug Costs $272,000 a Year. Not So Fast, Says New York State". The New York Times. ISSN 0362-4331. Retrieved 3 February 2019.
  49. ^ Johnson, Lisa (25 July 2018). "Cystic fibrosis patients sue health ministers for coverage of expensive drug for rare disease". CBC News. Retrieved 3 February 2019.
  50. ^ Boseley, Sarah (3 February 2019). "Cystic fibrosis: the patients facing shorter lives due to drug's expense". The Guardian. ISSN 0261-3077. Retrieved 3 February 2019.
  51. ^ "CF Support Group | Letter to Dr Jeffrey Leiden". CF Support Group. Retrieved 3 February 2019.
  52. ^ "The problem is the price: 5 reasons the cystic fibrosis community and its allies must band together to speak out about our drug prices – right now". Patients For Affordable Drugs. 17 May 2018. Retrieved 3 February 2019.
  53. ^ Boseley, Sarah (27 March 2019). "600 years' supply of cystic fibrosis drug destroyed in price row". the Guardian. Retrieved 29 March 2019.
  54. ^ "Nearly 8,000 Packets of Orkambi Destroyed as the U.K. and Vertex Hash out Pricing Concerns". BioSpace. 28 March 2019. Retrieved 19 June 2019.
  55. ^ Silverman, Ed (24 October 2019). "Vertex and the U.K. reach a deal for cystic fibrosis medicines". STAT. Retrieved 29 October 2019.
  56. ^ "NHS England finally strikes deal for high value drug". Health Service Journal. 24 October 2019. Retrieved 3 December 2019.
  57. ^ MS, Marisa Wexler (1 July 2020). "NHS, Vertex Strike Deal to Make Kaftrio Available in England". Cystic Fibrosis News Today. Retrieved 9 July 2020.
  58. ^ Manalac, Tristan (21 June 2024). "Vertex Finally Reaches Pricing Deal With England's NHS for Cystic Fibrosis Drugs". BioSpace.
  59. ^ "Spanish Government Approves National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) in Combination With KALYDECO® (ivacaftor)". www.businesswire.com. 21 October 2019.
  60. ^ a b c Lobo, Andrea (26 April 2024). "Access to Trikafta almost exclusive to high-income countries: Study". Cystic Firbrosis News Today.
  61. ^ Yoganathan, Venilla (8 May 2024). "SA to import costly cystic fibrosis drug, but few can afford it". Juta MedicalBrief.
  62. ^ a b c Andrew Pollack (19 November 2014), "Deal by Cystic Fibrosis Foundation Raises Cash and Some Concern", The New York Times, retrieved 19 July 2015
  63. ^ Joseph Walker; Jonathan D. Rockoff (19 November 2014), Cystic Fibrosis Foundation Sells Drug's Rights for $3.3 Billion: The Biggest Royalty Purchase Ever Reflects Group's Share of Kalydeco Sales, The Wall Street Journal, retrieved 19 July 2015
  64. ^ Brady Dennis (2 July 2015), "Are risks worth the rewards when nonprofits act like venture capitalists?", The Washington Post, retrieved 19 July 2015
  65. ^ a b John Fauber (19 May 2013). "Cystic Fibrosis: Charity and Industry Partner for Profit". MedPage Today, Milwaukee Journal Sentinel. Retrieved 19 July 2015.
  66. ^ David M. Orenstein; Paul M. Quinton; Brian P. O'Sullivan; Carlos E. Milla; Mark Pian; et al. (9 July 2012), Letter to Jeff Leiden, CEO of Vertex Pharmaceuticals (PDF), retrieved 19 July 2015
  67. ^ "Voices Carry: How History Informs Patient Advocacy in the Biotech Industry", Life Sciences Foundation, Cambridge, Massachusetts, 15 April 2015, archived from the original on 7 March 2016, retrieved 19 July 2015
  68. ^ "Voices Carry: How History Informs Patient Advocacy in the Biotech Industry". YouTube. 14 May 2014. 1 hour 3 min – 1 hour 5 min. Archived from the original on 13 December 2021. Retrieved 3 July 2019.
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