• Paul Negulescu, Fredrick Van Goor and Sabine Hadida receive Breakthrough Prize for developing transformative medicines for people with cystic fibrosis

BOSTON / Sep 14, 2023 / Business Wire / Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that three of its researchers have been awarded the 2024 Breakthrough Prize in Life Sciences “for developing life-transforming drug combinations that repair the defective chloride channel protein in patients with cystic fibrosis.” Specifically, the award celebrates the work of Paul Negulescu, Ph.D., Fredrick Van Goor, Ph.D., and Sabine Hadida, Ph.D., who have worked together to lead cystic fibrosis (CF) discovery for over 20 years.

“The research teams led by Paul, Fred and Sabine discovered the first and only medicines that address the underlying cause of cystic fibrosis. This remarkable effort required discovery of three novel mechanisms of action, resulting in the first disease modifying therapies that act by restoring function to a misfolded protein,” said David Altshuler, M.D., Ph.D. Executive Vice President, Global Research, and Chief Scientific Officer. “This prize recognizes the dedication and creativity of thousands of people at Vertex spanning research, development, regulatory, manufacturing and patient access, as well as our partners in the CF community, who have worked tirelessly to bring these medicines to people with CF around the world.”

The gene responsible for cystic fibrosis was discovered in 1989, but until the work of Vertex scientists, treatment of CF addressed only its symptoms rather than the underlying cause of the disease. Today, Vertex’s four approved oral medicines treat CF by improving the function of the defective CFTR protein. The first of these medicines, KALYDECO^® (ivacaftor), was approved in 2012, and today is approved for people with CF ages 1 month and older carrying responsive CFTR mutations. The most recently developed medicine, TRIKAFTA^®, is a triple combination therapy (elexacaftor/tezacaftor/ivacaftor and ivacaftor) first approved in 2019 that today can treat ~90% of people with CF who have responsive mutations.

Vertex is currently developing a next-in-class investigational CF triple combination therapy, now in Phase 3 trials, that has the potential for enhanced clinical benefit. The company is also in early clinical trials in collaboration with Moderna to develop an mRNA therapeutic designed to treat the approximately 5,000 people with CF who do not produce any CFTR protein. If this mRNA therapy is successful, it will be possible for all people with CF to have medicines to treat the underlying cause of their disease.

The Breakthrough Prize in Life Sciences was founded in 2013 by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Yuri and Julia Milner, and Anne Wojcicki. Breakthrough Prizes are also awarded annually in the fields of Fundamental Physics and Mathematics. To learn more, visit breakthroughprize.org.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.

About TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways.

About KALYDECO^® (ivacaftor)

In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. KALYDECO^® (ivacaftor) was the first medicine to treat the underlying cause of cystic fibrosis in people with specific mutations in the CFTR gene.

U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 2 years of age.

IMPORTANT SAFETY INFORMATION

Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: are allergic to TRIKAFTA or any ingredients in TRIKAFTA, have kidney problems, have or have had liver problems, are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby, or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk.

Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Patients should ask their doctor or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their doctor if they take: antibiotics such as rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. John’s wort; antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; antibiotics including telithromycin, clarithromycin, or erythromycin.

Patients should avoid food or drink that contains grapefruit while taking TRIKAFTA.

TRIKAFTA can cause serious side effects, including:

Liver damage and worsening of liver function in patients with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in patients without liver disease.

High liver enzymes in the blood, which is a common side effect in patients treated with TRIKAFTA. These can be serious and may be a sign of liver injury. The patient’s doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine.

Serious allergic reactions have happened to patients who are treated with TRIKAFTA. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include: rash or hives; tightness of the chest or throat or difficulty breathing; swelling of the face, lips and/or tongue; difficulty swallowing; and light-headedness or dizziness.

Abnormality of the eye lens (cataract) has been noted in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts.

The most common side effects of TRIKAFTA include headache, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, flu (influenza), inflamed sinuses, and increase in blood bilirubin.

Patients should tell their doctor if they have any side effect that bothers them or that does not go away. These are not all the possible side effects of TRIKAFTA. For more information, patients should ask their doctor or pharmacist.

Please click here to see the full Prescribing Information for TRIKAFTA.

U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO^® (ivacaftor)

INDICATIONS AND USAGE

KALYDECO (ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 1 month of age.

IMPORTANT SAFETY INFORMATION

Before taking KALYDECO, patients should tell their doctor about all their medical conditions, including if they: have liver or kidney problems; are allergic to KALYDECO or any ingredients; are pregnant or plan to become pregnant because it is not known if KALYDECO will harm an unborn baby; and are breastfeeding or planning to breastfeed because is not known if KALYDECO passes into breast milk.

Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Patients should ask their doctor or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their doctor if they take the antibiotics rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. John’s wort; antifungal medicines such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.

KALYDECO can cause dizziness in some patients who take it. If patients experience dizziness, they should not drive or operate machines until symptoms improve.

Patients should avoid food or drink containing grapefruit while taking KALYDECO.

KALYDECO can cause serious side effects including:

High liver enzymes in the blood, which have happened in patients receiving KALYDECO. The patient’s doctor will do blood tests to check their liver before starting KALYDECO, every 3 months during the first year of taking KALYDECO, and every year while taking KALYDECO. For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often.

Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.

Serious allergic reactions have happened to patients who are treated with KALYDECO. Patients should call their healthcare provider or go to the emergency room right away if they have symptoms of an allergic reaction. Symptoms of an allergic reaction may include rash or hives, tightness of the chest or throat or difficulty breathing, and light-headedness or dizziness.

Abnormality of the eye lens (cataract), which has happened in some children and adolescents receiving KALYDECO. The patient’s doctor should perform eye examinations before and during treatment with KALYDECO to look for cataracts.

The most common side effects include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

Use of KALYDECO in patients aged 1 month to less than 6 months born from a pregnancy lasting (gestational age) less than 37 weeks has not been evaluated.

These are not all the possible side effects of KALYDECO. Please click here to see the full Prescribing Information for KALYDECO.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, mRNA, cell and genetic therapies (including gene editing) in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 13 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding our plans and expectations for our pipeline, including our next-in-class investigational CF triple combination therapy and our mRNA therapy in collaboration with Moderna. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company’s studies may not be indicative of final clinical trial results, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, or other reasons, that our development programs may experience delays, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent filings filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.