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1. 24 February 2021 Vertex to Present at the Cowen Health Care Conference on March 2

   Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Cowen Health Care Conference on Tuesday, March 2, 2021 at 9:50 a.m. ET.

   The audio portion of management's remarks will be available live through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.

   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…

   Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Cowen Health Care Conference on Tuesday, March 2, 2021 at 9:50 a.m. ET.

   The audio portion of management's remarks will be available live through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.

   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 pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

   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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. 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.

   (VRTX-WEB)

   

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2. 09 February 2021 Aquestive Therapeutics Strengthens Board of Directors with Appointments of Julie Krop, M.D., and Marco Taglietti, M.D., and Announces Resignation of Douglas K. Bratton from Board of Directors

   WARREN, N.J., Feb. 09, 2021 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ:AQST), a pharmaceutical company focused on developing and commercializing differentiated products that address patients' unmet needs and solve therapeutic problems, today announced the appointment of Julie Krop, M.D., Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN), and Marco Taglietti, M.D., Director, President and Chief Executive Officer of SCYNEXIS (NASDAQ:SCYX), to the Board of Directors of the Company effective February 10, 2021. Aquestive also announced the resignation of Douglas K. Bratton from the Board of Directors after more than 17 years of service. Aquestive's Board of Directors will now be comprised of eight Directors, seven of whom…

   WARREN, N.J., Feb. 09, 2021 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ:AQST), a pharmaceutical company focused on developing and commercializing differentiated products that address patients' unmet needs and solve therapeutic problems, today announced the appointment of Julie Krop, M.D., Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN), and Marco Taglietti, M.D., Director, President and Chief Executive Officer of SCYNEXIS (NASDAQ:SCYX), to the Board of Directors of the Company effective February 10, 2021. Aquestive also announced the resignation of Douglas K. Bratton from the Board of Directors after more than 17 years of service. Aquestive's Board of Directors will now be comprised of eight Directors, seven of whom are independent. Dr. Krop will serve as a member of the Board's Nominating and Corporate Governance Committee and Dr. Taglietti will serve as a member of the Board's Audit Committee.
   
   

   "We are delighted to welcome Julie and Marco as new independent directors to the Aquestive Board of Directors," said Santo Costa, Aquestive's Chairman of the Board. "Their significant expertise in the pharmaceutical and biotechnology industry and impressive backgrounds complement our Board of Directors' skills and experiences. We are confident they will provide valuable perspectives as we continue to execute our strategy and enhance value for the Company's shareholders."

   Dr. Krop stated, "I am thrilled to join the Board of Directors of Aquestive and look forward to working with the Board and the leadership team to advance the Company's mission of providing novel alternatives to invasively administered standard of care therapies. I am particularly excited about helping management guide Aquestive's proprietary orally administered epinephrine through clinical development. This novel formulation of epinephrine has the potential to eliminate the burden of intramuscular or subcutaneous injections for patients at risk of anaphylaxis due to severe allergies."

   Dr. Taglietti commented, "Aquestive is at an important stage of its evolution and I am delighted to join the Board of Directors during this exciting time. I look forward to contributing to Aquestive's continued growth and success as the Company advances its Libervant™ (diazepam) Buccal Film application through approval and executes on its innovative development activities with the potential to change patients' lives."

   "Julie and Marco join Aquestive at an exciting time as we continue to focus on developing and bringing to market valuable products in the CNS and allergy spaces. Julie and Marco's extensive collective experience in leading clinical development, regulatory strategies and commercialization of key value assets is a great addition and complement to the skills already present on our board," remarked Keith Kendall, Director, President and Chief Executive Officer of Aquestive. "We've experienced significant growth and strengthened our capabilities as a commercial pharmaceutical company under Doug's leadership as the original Chairman of the Board and then director. I would like to thank him for his contributions and expert guidance that have well positioned Aquestive for continued momentum and success."

   "On behalf of the Board of Directors and the Company, I would like to thank Doug for his more than 17 years of service to our Board, including as former Chairman, in guiding the Company from its start-up phase to a public commercial pharmaceutical company and leader in the film-based therapeutics industry," said Mr. Costa.

   "It has been a great privilege to serve as a director and former Chairman of Aquestive's Board of Directors, the members of which I hold in very high regard. I'd like to thank Keith, his team, and the Board for many exciting and satisfying years, and look forward to watching them accomplish even more great things for Aquestive in the months and years to come," stated Mr. Bratton.

   About Douglas K. Bratton
   
   Mr. Bratton has served as a member of the Company's Board of Directors since January 2004 and was the Chairman of the Board from January 2004 until August 2018. Mr. Bratton is the Founder, President and Chief Investment Officer of Crestline Investors, an institutional alternative investment management firm. He has been an investment professional specializing in alternative asset strategies since 1983 and has managed assets on behalf of the Bass family of Fort Worth, Texas since 1988.

   About Julie Krop, M.D.
   
   Dr. Krop is a seasoned biotech executive with more than two decades of experience successfully designing and executing clinical development programs from early stage development all the way through FDA approval. She has held senior leadership roles across clinical development, regulatory affairs, clinical operations, pharmacovigilance, medical affairs and program management during her career in the pharmaceutical and biotechnology industry. She currently serves as the Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN). Prior to assuming her position with Freeline Therapeutics, Dr. Krop was the Chief Medical Officer and Executive Vice President, Development, at AMAG Pharmaceuticals (NASDAQ:AMAG) from 2015 to 2020. From 2012 to 2015, Dr. Krop served as the Vice President, Clinical Development for Vertex Pharmaceuticals (NASDAQ:VRTX). In addition, Dr. Krop was Vice President, Clinical Development and Regulatory Affairs for Stryker Biotech (NYSE:SYK) from 2006 to 2012. Dr. Krop received a B.A. from Brown University and her M.D. from Brown University School of Medicine. She completed her fellowship in the Department of Endocrinology at the Johns Hopkins University School of Medicine where she was also a Robert Wood Johnson Foundation Clinical Scholar.

   About Marco Taglietti, M.D.
   
   Dr. Taglietti has more than three decades of experience in the pharmaceutical and biotechnology industry. He currently serves as a Director and President and Chief Executive Officer of SCYNEXIS Inc. (NASDAQ:SCYX). Prior to joining SCYNEXIS, Dr. Taglietti held various executive positions with Forest Laboratories (now AbbVie (NYSE: ABBV)) from 2007 until 2014, including President, Forest Research Institute, Chief Medical Officer and Executive Corporate Vice President, Research & Development. Dr. Taglietti was also the Senior Vice President, Head of Global Research and Development for Stiefel Laboratories, Inc. (now a GlaxoSmithKline company) from 2004 until 2007 and served in a number of executive positions from 1992 to 2004 with Schering-Plough Research Institute, including Vice President, Clinical Research Anti-Infectives, CNS, Dermatology and Endocrinology. From 1987 until 1992, Dr. Taglietti served in a number of executive positions with Marion Merrell Dow Research Institute, including as the European Product Team Leader – Anti-Infectives. Dr. Taglietti previously served on the boards of directors of Delcath (NASDAQ:DCTH) from 2014 to 2020 and NephroGenex (NASDAQ:NRX) from 2014 to 2017. Dr. Taglietti also served as a director of Stiefel International, Ltd., a private company, from 2004 to 2007 and a director of TransCelerate BioPharma, a non-profit pharma coalition dedicated to streamlining and accelerating the research and development of innovative new therapies, from 2013 to 2014. Since 2011, Dr. Taglietti has served on the board of directors of BioNJ, a life sciences trade association in New Jersey. In addition, Dr. Taglietti served on the board of directors of HINJ, Health Institute of New Jersey, a trade association for the leading research-based biopharmaceutical and medical technology companies in New Jersey, from 2011 to 2014, and is currently on the boards of directors of Orchestra of St. Luke, a New York City based orchestra, and American Foundation for Suicide Prevention, the largest non-profit organization dedicated to saving lives and bringing hope to those affected by suicide. Dr. Taglietti received his Degree in Medicine from the University of Pavia, Italy.

   About Aquestive Therapeutics
   
   Aquestive Therapeutics is a pharmaceutical company that applies innovative technology to solve therapeutic problems and improve medicines for patients. The Company has commercialized one internally-developed proprietary product to date, Sympazan® (clobazam) oral film, has a commercial proprietary product pipeline focused on the treatment of diseases of the central nervous system, or CNS, and other unmet needs, and is developing orally administered complex molecules to provide alternatives to invasively administered standard of care therapies. The Company also collaborates with other pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven capabilities for drug development and commercialization.

   Forward-Looking Statements
   
   Certain statements in this press release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "believe," "anticipate," "plan," "expect," "estimate," "intend," "may," "will," or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding the advancement of Libervant and other product candidates through the regulatory and development pipeline; and business strategies, market opportunities, and other statements that are not historical facts. These forward-looking statements are subject to the uncertain impact of the COVID-19 global pandemic on our business including with respect to our clinical trials including site initiation, patient enrollment and timing and adequacy of clinical trials; on regulatory submissions and regulatory reviews and approvals of our product candidates; pharmaceutical ingredient and other raw materials supply chain, manufacture, and distribution; sale of and demand for our products; our liquidity and availability of capital resources; customer demand for our products and services; customers' ability to pay for goods and services; and ongoing availability of an appropriate labor force and skilled professionals. Given these uncertainties, the Company is unable to provide assurance that operations can be maintained as planned prior to the COVID-19 pandemic.

   These forward-looking statements are based on our current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks and uncertainties include, but are not limited to, risks associated with the Company's development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and plans for AQST-108 and our other drug candidates; risk of delays in FDA approval of our drug candidate Libervant and AQST-108 and our other drug candidates or failure to receive approval; ability to address the concerns identified in the FDA's Complete Response Letter dated September 25, 2020 regarding the New Drug Application for Libervant; risk of our ability to demonstrate to the FDA "clinical superiority" within the meaning of the FDA regulations of Libervant relative to FDA-approved diazepam rectal gel and nasal spray products including by establishing a major contribution to patient care within the meaning of FDA regulations relative to the approved products as well as risks related to other potential pathways or positions which are or may in the future be advanced to the FDA to overcome the seven year orphan drug exclusivity granted by the FDA for the approved nasal spray product of a competitor in the U.S. and there can be no assurance that we will be successful; risk that a competitor obtains FDA orphan drug exclusivity for a product with the same active moiety as any of our other drug products for which we are seeking FDA approval and that such earlier approved competitor orphan drug blocks such other product candidates in the U.S. for seven years for the same indication; risk inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks and regulatory limitations); risks and uncertainties concerning the royalty and other revenue stream of the KYNMOBI™ monetization transaction, achievement of royalty targets worldwide or in any jurisdiction and certain other commercial targets required for contingent payments under the monetization transaction, and of sufficiency of net proceeds of the monetization transaction after satisfaction of and compliance with 12.5% Senior Notes obligations, as applicable, and for funding the Company's operations; risk of development of our sales and marketing capabilities; risk of legal costs associated with and the outcome of our patent litigation challenging third party at risk generic sale of our proprietary products; risk of sufficient capital and cash resources, including access to available debt and equity financing and revenues from operations, to satisfy all of our short-term and longer term cash requirements and other cash needs, at the times and in the amounts needed; risk of failure to satisfy all financial and other debt covenants and of any default; our and our competitors' orphan drug approval and resulting drug exclusivity for our products or products of our competitors; short-term and long-term liquidity and cash requirements, cash funding and cash burn; risk related to government claims against Indivior for which we license, manufacture and sell Suboxone® and which accounts for the substantial part of our current operating revenues; risk associated with Indivior's cessation of production of its authorized generic buprenorphine naloxone film product, including the impact from loss of orders for the authorized generic product and risk of eroding market share for Suboxone and risk of sunsetting product; risks related to the outsourcing of certain marketing and other operational and staff functions to third parties; risk of the rate and degree of market acceptance of our product and product candidates; the success of any competing products, including generics; risk of the size and growth of our product markets; risks of compliance with all FDA and other governmental and customer requirements for our manufacturing facilities; risks associated with intellectual property rights and infringement claims relating to the Company's products; risk of unexpected patent developments; the impact of existing and future legislation and regulatory provisions on product exclusivity; legislation or regulatory actions affecting pharmaceutical product pricing, reimbursement or access; claims and risks that may arise regarding the safety or efficacy of the Company's products and product candidates; risk of loss of significant customers; risks related to legal proceedings, including patent infringement, investigative and antitrust litigation matters; changes in government laws and regulations; risk of product recalls and withdrawals; uncertainties related to general economic, political, business, industry, regulatory and market conditions and other unusual items; and other uncertainties affecting the Company described in the "Risk Factors" section and in other sections included in our Annual Report on Form 10 K, in our Quarterly Reports on Form 10-Q, and in our Current Reports on Form 8-K filed with the Securities Exchange Commission (SEC). Given those uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date made. All subsequent forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. The Company assumes no obligation to update forward-looking statements or outlook or guidance after the date of this press release whether as a result of new information, future events or otherwise, except as may be required by applicable law.

   PharmFilm®, Sympazan® and the Aquestive logo are registered trademarks of Aquestive Therapeutics, Inc. All other registered trademarks referenced herein are the property of their respective owners.

   Investor inquiries:
   
   Westwicke, an ICR Company
   
   Stephanie Carrington
   
   
   
   646-277-1282

    
   
   

   
   
   

   

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3. 01 February 2021 Vertex Reports Full-Year and Fourth-Quarter 2020 Financial Results

   -Full-year 2020 GAAP product revenues of $6.20 billion-

   -Full-year 2020 non-GAAP product revenues of $6.20 billion, a 55% increase compared to full-year 2019-

   -Company provides full-year 2021 product revenue guidance of $6.7 to $6.9 billion-

   Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the full year and fourth quarter ended December 31, 2020 and provided full-year 2021 financial guidance.

   "Our achievements in 2020 were marked by a significant increase in the number of people treated with the triple combination in the U.S. and the EU. It was also a year marked by meaningful pipeline advancement. We now have clinical programs in seven disease areas, spanning multiple modalities including…

   -Full-year 2020 GAAP product revenues of $6.20 billion-

   -Full-year 2020 non-GAAP product revenues of $6.20 billion, a 55% increase compared to full-year 2019-

   -Company provides full-year 2021 product revenue guidance of $6.7 to $6.9 billion-

   Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the full year and fourth quarter ended December 31, 2020 and provided full-year 2021 financial guidance.

   "Our achievements in 2020 were marked by a significant increase in the number of people treated with the triple combination in the U.S. and the EU. It was also a year marked by meaningful pipeline advancement. We now have clinical programs in seven disease areas, spanning multiple modalities including small molecules for alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases, gene editing for sickle cell disease and beta thalassemia, and cell therapy for type 1 diabetes," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "As we enter 2021, we look forward to treating more CF patients and reaching important R&D milestones in multiple additional diseases which will fuel our growth this year and for many years to come."

   Fourth-Quarter and Full-Year 2020 Financial Highlights

   		Three Months Ended December 31,								%			Twelve Months Ended December 31,								%
   		2020				2019				Change			2020				2019				Change
   		(in millions, except per share amounts)
   GAAP Product revenues, net		$	1,627			$	1,413			15	%		$	6,203			$	4,161			49	%
   Non-GAAP Product revenues, net (1)		$	1,627			$	1,257			29	%		$	6,203			$	4,005			55	%
   GAAP Operating income		$	746			$	551			35	%		$	2,856			$	1,198			139	%
   Non-GAAP Operating income		$	887			$	593			50	%		$	3,491			$	1,786			95	%
   GAAP Net income		$	604			$	583			4	%		$	2,712			$	1,177			130	%
   Non-GAAP Net income		$	661			$	444			49	%		$	2,719			$	1,389			96	%
   GAAP Net income per share - diluted		$	2.30			$	2.23			3	%		$	10.29			$	4.51			128	%
   Non-GAAP Net income per share - diluted		$	2.51			$	1.70			48	%		$	10.32			$	5.33			94	%

   Full-Year 2020 Results

   		Twelve Months Ended December 31,
   		2020				2019
   		(in millions)
   GAAP Product revenues, net		$	6,203			$	4,161
   Non-GAAP Product revenues, net (1)		$	6,203			$	4,005
   TRIKAFTA/KAFTRIO		$	3,864			$	420
   SYMDEKO/SYMKEVI		$	629			$	1,418
   ORKAMBI		$	908			$	1,176
   KALYDECO		$	803			$	991

   GAAP and Non-GAAP product revenues increased 49% and 55%, respectively, compared to 2019, primarily driven by the uptake of TRIKAFTA in the U.S., KAFTRIO in Europe and our other medicines outside the U.S. following the completion of several significant reimbursement agreements. Net product revenues in 2020 were $4.8 billion in the U.S. and $1.4 billion outside the U.S.

   GAAP and Non-GAAP net income increased compared to 2019, largely driven by strong growth in product revenues.

   Cash, cash equivalents and marketable securities as of December 31, 2020 were $6.7 billion, an increase of approximately $2.9 billion compared to $3.8 billion as of December 31, 2019 driven by strong revenue and profitability.

   Full-Year 2020 Expenses

   		Twelve Months Ended December 31,
   		2020				2019
   		(in millions)
   Combined GAAP R&D and SG&A expenses		$	2,600			$	2,413
   Combined Non-GAAP R&D and SG&A expenses		$	1,981			$	1,694
   GAAP R&D expense		$	1,830			$	1,755
   Non-GAAP R&D expense		$	1,372			$	1,171
   GAAP SG&A expense		$	770			$	658
   Non-GAAP SG&A expense		$	609			$	523
   GAAP income taxes		$	405			$	218
   Non-GAAP income taxes		$	721			$	397
   GAAP effective tax rate			13	%			16	%
   Non-GAAP effective tax rate (2)			21	%			22	%

   Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to 2019, primarily due to the incremental investment to support the global use of Vertex's medicines and the expansion of Vertex's pipeline in CF and other disease areas.

   GAAP and Non-GAAP income taxes increased compared to 2019 primarily due to Vertex's increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex's provision for income taxes.

   Fourth-Quarter 2020 Results

   		Three Months Ended December 31,
   		2020				2019
   		(in millions)
   GAAP Product revenues, net		$	1,627			$	1,413
   Non-GAAP Product revenues, net (1)		$	1,627			$	1,257
   TRIKAFTA/KAFTRIO		$	1,091			$	420
   SYMDEKO/SYMKEVI		$	128			$	332
   ORKAMBI		$	215			$	270
   KALYDECO		$	193			$	236

   GAAP and Non-GAAP product revenues increased 15% and 29%, respectively, compared to the fourth quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S., KAFTRIO in Europe and our other medicines outside the U.S. following the completion of several significant reimbursement agreements.

   GAAP and non-GAAP net income increased compared to the fourth quarter of 2019, largely driven by strong growth in total product revenues.

   Fourth-Quarter 2020 Expenses

   		Three Months Ended December 31,
   		2020				2019
   		(in millions)
   Combined GAAP R&D and SG&A expenses		$	678			$	675
   Combined Non-GAAP R&D and SG&A expenses		$	539			$	496
   GAAP R&D expense		$	467			$	480
   Non-GAAP R&D expense		$	364			$	337
   GAAP SG&A expense		$	212			$	195
   Non-GAAP SG&A expense		$	175			$	159
   GAAP income taxes		$	284			$	94
   Non-GAAP income taxes		$	198			$	145
   GAAP effective tax rate			32	%			14	%
   Non-GAAP effective tax rate (2)			23	%			25	%

   Combined GAAP R&D and SG&A expenses were similar to the fourth quarter of 2019.

   Combined Non-GAAP R&D and SG&A expenses increased compared to the fourth quarter of 2019, primarily due to the incremental investment to support the global use of Vertex's medicines and the expansion of Vertex's pipeline in CF and other disease areas.

   GAAP and Non-GAAP income taxes increased compared to the fourth quarter of 2019 primarily due to Vertex's increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex's provision for income taxes.

   Full-Year 2021 Financial Guidance

   Vertex today provided its full-year 2021 financial guidance. Product revenue guidance is primarily based on:

   • The continued strong performance of TRIKAFTA in the U.S. and KAFTRIO in certain European countries
   • The launch of medicines in the U.S. for rare mutations following approval in December 2020 and the approval of TRIKAFTA for children with CF ages 6-11 in the U.S. expected mid-year
   • Countries where patients currently have access or reimbursement

   Vertex's guidance is summarized below:

   		FY 2021
   Product revenues		$6.7 to 6.9 billion
   Combined GAAP R&D and SG&A expenses (3)		$2.9 to 3.05 billion
   Combined Non-GAAP R&D and SG&A expenses (3)		$2.25 to 2.3 billion
   Non-GAAP effective tax rate		21% to 22%

   Key Business Highlights

   Cystic Fibrosis (CF) R&D pipeline

   Vertex expects to increase the number of CF patients eligible to take our medicines and thereby continue to grow our CF business. Important progress has been made in increasing the number of eligible patients and expanding approval and access for our medicines to additional geographies and age groups.

   TRIKAFTA/KAFTRIO (elexacaftor, tezacaftor and ivacaftor)

   • The U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA to include people with CF ages 12 and older with certain mutations that are responsive to TRIKAFTA based on in vitro data. SYMDEKO and KALYDECO also received approvals to include additional responsive mutations in people with CF ages 6 and older and ages 4 months and older, respectively.
   • Swissmedic, the Swiss Agency for Therapeutic Products, granted marketing authorization and a reimbursement agreement was reached for TRIKAFTA in Switzerland in people with CF ages 12 and older with two F508del mutations or one F508del mutation and one minimal function mutation.
   • The U.S. FDA accepted a supplemental New Drug Application (sNDA) for TRIKAFTA for the treatment of children with CF ages 6 to 11 who have at least one F508del mutation or have certain mutations that are responsive to TRIKAFTA based on in vitro data. The FDA granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021.
   • Health Canada accepted a New Drug Submission for Priority Review for TRIKAFTA for the treatment of people with CF ages 12 years and older.

   SYMDEKO/SYMKEVI (tezacaftor and ivacaftor)

   • The European Commission (EC) granted approval of the label extension for SYMKEVI to include people with CF ages 6 years and older with two copies of the F508del mutation or one copy of the F508del mutation and certain residual function mutations.

   KALYDECO (ivacaftor)

   • The EC granted approval of the label extension for KALYDECO to include the treatment of infants with CF ages 4 months and older who have R117H or certain gating mutations.

   R&D pipeline outside of CF

   Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:

   Beta Thalassemia and Sickle Cell Disease

   • Vertex and its partner CRISPR Therapeutics are evaluating the use of an ex vivo CRISPR gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). This approach aims to edit a person's hematopoietic stem cells to produce fetal hemoglobin in red blood cells, which has the potential to reduce or eliminate symptoms associated with disease.
   • Enrollment and dosing are ongoing in the clinical studies for CTX001. More than 20 patients have been dosed with CTX001 across both studies to date. Completion of enrollment in both studies is expected in 2021.

   Alpha-1 Antitrypsin (AAT) Deficiency

   • Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency.
   • Enrollment is ongoing in a Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864. Data from this study is expected in the first half of 2021.

   APOL1-mediated Kidney Diseases

   • Vertex is evaluating the potential for inhibitors of APOL1 function to reduce proteinuria in people with serious kidney diseases, including focal segmental glomerulosclerosis (FSGS).
   • Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147. Data from this study is expected in 2021.

   Type 1 Diabetes (T1D)

   • Vertex is developing a cell therapy designed to replace insulin-producing islet cells in people with T1D. Two opportunities exist for the transplant of these functional islets into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device.
   • The U.S. FDA cleared the Investigational New Drug Application (IND) for VX-880, the islet cells alone program. Vertex expects to initiate a Phase 1/2 clinical trial in the first half of 2021.

   Investments in External Innovation

   • Skyhawk Therapeutics and Vertex established a strategic collaboration to discover and develop novel small molecules that modulate RNA splicing for the treatment of serious diseases.

   Non-GAAP Financial Measures

   In this press release, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex's pre-tax income (i) stock-based compensation expense, (ii) an adjustment to product revenues and related cost of sales to reflect the conclusion of the early access program for ORKAMBI in France in the fourth quarter of 2019, (iii) revenues and expenses related to collaboration agreements, (iv) gains or losses related to the fair value of the company's strategic investments, (v) increases or decreases in the fair value of contingent consideration, (vi) acquisition-related costs and (vii) other adjustments. The company's non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company's GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company's business, are important in comparing current results with prior period results and provide additional information regarding the company's financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company's business and to evaluate its performance. The company adjusts, where appropriate, for both revenues and expenses in order to reflect the company's operations. The company's calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

   The company provides guidance regarding combined R&D and SG&A expenses and effective tax rate on a non-GAAP basis. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities. The company does not provide a GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.

   Vertex Pharmaceuticals Incorporated
   Fourth-Quarter Results
   Consolidated Statements of Operations
   (in thousands, except per share amounts)
   (unaudited)
   		Three Months Ended December 31,								Twelve Months Ended December 31,
   		2020				2019				2020				2019
   Revenues:
   Product revenues, net		$	1,626,920			$	1,413,265			$	6,202,783			$	4,160,726
   Collaboration and royalty revenues		900				—				2,900				2,095
   Total revenues		1,627,820				1,413,265				6,205,683				4,162,821
   Costs and expenses:
   Cost of sales		203,101				185,012				736,300				547,758
   Research and development expenses		466,584				480,011				1,829,537				1,754,540
   Sales, general and administrative expenses		211,843				195,277				770,456				658,498
   Change in fair value of contingent consideration		500				1,500				13,100				4,459
   Total costs and expenses		882,028				861,800				3,349,393				2,965,255
   Income from operations		745,792				551,465				2,856,290				1,197,566
   Interest income		2,320				12,359				22,239				63,678
   Interest expense		(16,288		)		(14,249		)		(58,151		)		(58,502		)
   Other income, net (4)		156,799				127,375				296,420				192,177
   Income before provision for income taxes		888,623				676,950				3,116,798				1,394,919
   Provision for income taxes		284,433				93,716				405,151				218,109
   Net income		$	604,190			$	583,234			$	2,711,647			$	1,176,810
   Net income per common share:
   Basic		$	2.32			$	2.26			$	10.44			$	4.58
   Diluted		$	2.30			$	2.23			$	10.29			$	4.51
   Shares used in per share calculations:
   Basic		260,038				258,003				259,841				256,728
   Diluted		263,106				262,108				263,396				260,673

   Reconciliation of GAAP to Non-GAAP Net Income
   Fourth-Quarter Results
   (in thousands, except per share amounts)
   (unaudited)
   		Three Months Ended December 31,								Twelve Months Ended December 31,
   		2020				2019				2020				2019
   GAAP net income		$	604,190			$	583,234			$	2,711,647			$	1,176,810
   Stock-based compensation expense		97,027				91,591				429,461				360,489
   ORKAMBI Adjustment (1)		—				(140,854		)		—				(140,854		)
   Increase in fair value of strategic investments (4)		(171,071		)		(128,734		)		(311,937		)		(197,596		)
   Increase in fair value of contingent consideration (5)		500				1,500				13,100				4,459
   Collaborative revenues and expenses (6)		40,400				56,057				181,700				318,343
   Acquisition-related costs (7)		2,820				33,181				10,682				45,871
   Total non-GAAP adjustments to pre-tax income		(30,324		)		(87,259		)		323,006				390,712
   Tax adjustments (2)		86,728				(51,627		)		(315,455		)		(178,578		)
   Non-GAAP net income		$	660,594			$	444,348			$	2,719,198			$	1,388,944
   Net income per diluted common share:
   GAAP		$	2.30			$	2.23			$	10.29			$	4.51
   Non-GAAP		$	2.51			$	1.70			$	10.32			$	5.33
   Shares used in diluted per share calculations:
   GAAP and Non-GAAP		263,106				262,108				263,396				260,673

   Reconciliation of GAAP to Non-GAAP Revenues and Expenses
   Fourth-Quarter Results
   (in thousands)
   (unaudited)
   		Three Months Ended December 31,								Twelve Months Ended December 31,
   		2020				2019				2020				2019
   GAAP total revenues		$	1,627,820			$	1,413,265			$	6,205,683			$	4,162,821
   ORKAMBI Adjustment (1)		—				(155,773		)		—				(155,773		)
   Collaborative revenues		(900		)		—				(2,900		)		(158		)
   Non-GAAP total revenues		$	1,626,920			$	1,257,492			$	6,202,783			$	4,006,890
   		Three Months Ended December 31,								Twelve Months Ended December 31,
   		2020				2019				2020				2019
   GAAP cost of sales		$	203,101			$	185,012			$	736,300			$	547,758
   ORKAMBI Adjustment (1)		—				(14,919		)		—				(14,919		)
   Stock-based compensation expense		(1,581		)		(1,397		)		(5,579		)		(5,575		)
   Non-GAAP cost of sales		$	201,520			$	168,696			$	730,721			$	527,264
   GAAP research and development expenses		$	466,584			$	480,011			$	1,829,537			$	1,754,540
   Stock-based compensation expense		(58,958		)		(56,707		)		(262,690		)		(224,558		)
   Collaborative expenses (6)		(41,300		)		(56,057		)		(184,600		)		(318,501		)
   Acquisition-related costs (7)		(2,820		)		(30,461		)		(10,229		)		(40,583		)
   Non-GAAP research and development expenses		$	363,506			$	336,786			$	1,372,018			$	1,170,898
   GAAP sales, general and administrative expenses		$	211,843			$	195,277			$	770,456			$	658,498
   Stock-based compensation expense		(36,488		)		(33,487		)		(161,192		)		(130,356		)
   Acquisition-related costs (7)		—				(2,720		)		(453		)		(5,288		)
   Non-GAAP sales, general and administrative expenses		$	175,355			$	159,070			$	608,811			$	522,854
   Combined non-GAAP R&D and SG&A expenses		$	538,861			$	495,856			$	1,980,829			$	1,693,752
   		Three Months Ended December 31,								Twelve Months Ended December 31,
   		2020				2019				2020				2019
   GAAP other income, net		$	156,799			$	127,375			$	296,420			$	192,177
   Increase in fair value of strategic investments (4)		(171,071		)		(128,734		)		(311,937		)		(197,596		)
   Non-GAAP other expense, net		$	(14,272	)		$	(1,359	)		$	(15,517	)		$	(5,419	)
   GAAP provision for income taxes		$	284,433			$	93,716			$	405,151			$	218,109
   Tax adjustments (2)		(86,728		)		51,627				315,455				178,578
   Non-GAAP provision for income taxes (8)		$	197,705			$	145,343			$	720,606			$	396,687

   Condensed Consolidated Balance Sheets
   (in thousands)
   (unaudited)
   		December 31, 2020				December 31, 2019
   Assets
   Cash, cash equivalents and marketable securities		$	6,658,897			$	3,808,294
   Accounts receivable, net		885,352				633,518
   Inventories		280,777				167,502
   Property and equipment, net		958,534				745,080
   Goodwill and intangible assets		1,402,158				1,402,158
   Deferred tax assets		882,779				1,190,815
   Other assets		683,311				371,098
   Total assets		$	11,751,808			$	8,318,465
   Liabilities and Shareholders' Equity
   Accounts payable and accrued expenses		$	1,560,110			$	1,204,522
   Finance lease liabilities		581,476				577,371
   Contingent consideration		189,600				176,500
   Other liabilities		733,807				274,828
   Shareholders' equity		8,686,815				6,085,244
   Total liabilities and shareholders' equity		$	11,751,808			$	8,318,465
   Common shares outstanding		259,890				258,993

   Supplemental Income Tax Information
   (in thousands, except percentages)
   (unaudited)
   		Three Months Ended December 31,								Twelve Months Ended December 31,
   		2020				2019				2020				2019
   Components of provision for income taxes related to:
   Cash paid or accrued for income taxes		$	122,975			$	9,185			$	168,322			$	32,138
   Benefits from income taxes due to discrete tax items (2)		10,034				—				(287,565		)		—
   Provision for income taxes offset by net operating losses and credits (8)		151,424				84,531				524,394				185,971
   GAAP provision for income taxes (8)		$	284,433			$	93,716			$	405,151			$	218,109
   Cash paid or accrued for income taxes		$	122,975			$	9,185			$	168,322			$	32,138
   Adjustments to pre-tax income		(76,694		)		51,627				27,890				178,578
   Provision for income taxes offset by net operating losses and credits (8)		151,424				84,531				524,394				185,971
   Non-GAAP provision for income taxes (8)		$	197,705			$	145,343			$	720,606			$	396,687
   Effective tax rate reconciliation:
   GAAP effective tax rate		32		%		14		%		13		%		16		%
   Impact of GAAP to Non-GAAP adjustments		(9		)%		11		%		8		%		6		%
   Non-GAAP effective tax rate		23		%		25		%		21		%		22		%

   Notes and Explanations

   1: "ORKAMBI adjustment" in the company's Reconciliation of GAAP to Non-GAAP Net Income in the three and twelve months ended December 31, 2019 included an adjustment to net product revenues and cost of sales related to the conclusion of the early access program for ORKAMBI in France in the fourth quarter of 2019. The company had previously recognized a portion of net product revenues related to ORKAMBI distributed through the early access program in France. As a result, the company recognized an adjustment to increase net product revenues by $155.8 million and cost of sales by $14.9 million, which related to prior period shipments of ORKAMBI distributed through the early access program in France. The company excluded the adjustment to net product revenues and cost of sales from its Non-GAAP measures for the three and twelve months ended December 31, 2019.

   2: In the three and twelve months ended December 31, 2020 and 2019, "Tax adjustments" primarily related to the estimated income taxes related to non-GAAP adjustments to pre-tax income including (i) stock-based compensation (including an adjustment for excess tax benefits related to stock-based compensation), (ii) increases or decreases in the fair value of the company's strategic investments and (iii) collaborative payments. In the twelve months ended December 31, 2020, "Tax adjustments" also included non-recurring discrete benefits to the company's provision for income taxes, such as the transfer of intellectual property rights to the company's U.K. entity, of approximately $287.6 million that the company excluded from its Non-GAAP measures.

   3: The difference between the company's full-year 2021 combined GAAP R&D and SG&A expenses and combined non-GAAP R&D and SG&A expenses guidance relates primarily to $430 million to $500 million of stock-based compensation expense and $200 million to $250 million of R&D expenses related to existing collaboration agreements. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities.

   4: "Other income, net" includes gains related to changes in the fair value of the company's strategic investments and from sales of certain investments.

   5: During the three and twelve months ended December 31, 2020 and 2019, the increase in the fair value of contingent consideration relates to potential payments to Exonics Therapeutics' former equity holders.

   6: "Collaborative revenues and expenses" in the three and twelve months ended December 31, 2020 and 2019 primarily related to collaborative upfront and milestone payments.

   7: "Acquisition-related costs" in the three and twelve months ended December 31, 2020 and 2019 related to costs associated with the company's acquisitions of Semma Therapeutics and Exonics.

   8: The company records a provision for income taxes on its pre-tax income using an effective tax rate approximating statutory rates. Since the company released its valuation allowance on the majority of its net operating losses and other deferred tax assets as of December 31, 2018, its tax provision has included a significant non-cash charge due to the company's ability to offset its pre-tax income against previously benefited net operating losses and credits. As of December 31, 2019, the company had federal net operating losses and credits that were available to offset future pre-tax income. The company utilized substantially all of its remaining federal net operating losses in 2020. As a result, a larger portion of the company's tax provision will represent a cash expense in future periods, subject to continued utilization of certain tax credits.

   Note: Amounts may not foot due to rounding.

   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 pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

   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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. 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, including, without limitation, Dr. Kewalramani's statements in this press release, the information provided regarding future financial performance and operations, the section captioned "Full-Year 2021 Financial Guidance" and statements regarding (i) anticipated regulatory filings and data submissions, (ii) anticipated future label expansions, (iii) the expectations, development plans and anticipated timelines for the company's medicines, drug candidates and pipeline programs, including collaborations with third parties, (iv) expectations for the collaborations with CRISPR, including expectations regarding completion of enrollment, (v) expectations for uptake of and expanded access to the company's medicines, including additional reimbursement agreements, and (vi) anticipated investment in internal and external innovation. 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 the company's expectations regarding its 2021 product revenues, expenses and effective tax rates may be incorrect (including because one or more of the company's assumptions underlying its expectations may not be realized), that COVID-19 may have different or more significant impacts on the company's business or operations than the company currently expects, data from preclinical testing or early clinical trials, especially if based on a limited number of patients, may not be indicative of final results, that the FDA may not approve VX-880 on a timely basis, or at all, that data from the company's development programs may not support registration or further development of its potential medicines in a timely manner, or at all, due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and on the SEC's website at www.sec.gov. 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.

   Conference Call and Webcast

   The company will host a conference call and webcast today at 4:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section under "Events and Presentations." To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website.

   (VRTX-E)

   

   View source version on businesswire.com: https://www.businesswire.com/news/home/20210201005809/en/

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4. 28 January 2021 Vertex Announces FDA Clearance of Investigational New Drug (IND) Application for VX-880, a Novel Cell Therapy for the Treatment of Type 1 Diabetes (T1D)

   - Vertex will initiate a Phase 1/2 clinical trial in first half of 2021 -

   - VX-880 is the first stem cell-derived therapy evaluating fully differentiated pancreatic islet cells for the treatment of T1D -

   Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND, enabling the company to proceed with initiating a clinical trial for VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell therapy to treat T1D. Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2021 in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.

   "As we celebrate the 100^th anniversary of the discovery of insulin…

   - Vertex will initiate a Phase 1/2 clinical trial in first half of 2021 -

   - VX-880 is the first stem cell-derived therapy evaluating fully differentiated pancreatic islet cells for the treatment of T1D -

   Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND, enabling the company to proceed with initiating a clinical trial for VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell therapy to treat T1D. Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2021 in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.

   "As we celebrate the 100^th anniversary of the discovery of insulin this year, we are excited to bring a first-in-class cell therapy to the clinic with the potential to meaningfully impact people living with T1D," said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. "We look forward to getting our clinical program underway and testing our unique approach of replacing pancreatic islet cells, which are destroyed in people with type 1 diabetes, with our stem cell-derived fully differentiated insulin-producing pancreatic islet cells."

   About VX-880
   
   VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy that is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the body's ability to regulate glucose levels by restoring pancreatic islet cell function, including insulin production.

   The VX-880 clinical trial will involve an infusion of fully differentiated, functional islet cells, as well as the chronic administration of concomitant immunosuppressive therapy, to protect the islet cells from immune rejection.

   About the Phase 1/2 Clinical Trial
   
   The clinical trial is a Phase 1/2, single-arm, open-label study in subjects who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This will be a sequential, multi-part clinical trial to evaluate the safety and efficacy of different doses of VX-880. Approximately 17 patients will be enrolled in the clinical trial.

   About Type 1 Diabetes
   
   T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death. Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in patients with T1D. Hypoglycemia remains a critical limiting factor in glycemic management, and severe hypoglycemia can cause loss of consciousness, coma, seizures, injury, and can be fatal.

   There are currently limited treatment options beyond insulin for the management of T1D.

   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 pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

   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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. 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, including, without limitation, statements made by Bastiano Sanna, Ph.D., in this press release, statements regarding the development, plans and expectations for our T1D pipeline program, including our plans to initiate a Phase 1/2 clinical trial in people with T1D and expected timeline of our clinical trials, statements regarding patient enrollment and dosing, statements regarding potential clinical trial results and anticipated benefits of VX-880, and our plans to provide further updates on our T1D pipeline program. 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 the FDA may not approve our IND, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs may not support registration or further development due to safety, efficacy or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trials, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports 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.

   (VRTX-GEN)

   

   View source version on businesswire.com: https://www.businesswire.com/news/home/20210128006088/en/

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5. 26 January 2021 Vertex Announces U.S. FDA Acceptance of Supplemental New Drug Application for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations

   -FDA grants Priority Review of the application and sets a PDUFA target action date of June 8, 2021-

   Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) to expand the use of TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021. The submission was supported…

   -FDA grants Priority Review of the application and sets a PDUFA target action date of June 8, 2021-

   Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) to expand the use of TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021. The submission was supported by data from a global Phase 3 study of TRIKAFTA in children ages 6 through 11 years old with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.

   "If approved for this expanded use, we will have the opportunity to treat the underlying cause of the disease earlier in life with TRIKAFTA and potentially benefit approximately 1,500 additional children with CF," said Carmen Bozic, M.D., Executive Vice President and Chief Medical Officer at Vertex. "Since our initial approval of TRIKAFTA in 2019, we have continued to work tirelessly to bring this medicine to those waiting as quickly as possible. We look forward to working with the Agency as they review the application over the course of the coming months."

   Vertex plans to submit a Marketing Authorization Application (MAA) variation for the triple combination in the European Union in the first half of 2021 for children ages 6 through 11. Global regulatory filings in additional markets, including Canada and Australia, are planned in the coming months for this age group.

   About Cystic Fibrosis

   Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas 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. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins 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 that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

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

   What is TRIKAFTA?

   TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 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 12 years of age.

   Patients should not take TRIKAFTA if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. John's wort.

   Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: 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.

   TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. Therefore, the dose of TRIKAFTA may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.

   TRIKAFTA can cause dizziness in some people who take it. Patients should not drive a car, operate machinery, or do anything that needs them to be alert until they know how TRIKAFTA affects them.

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

   TRIKAFTA can cause serious side effects, including:

   High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. 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.

   Abnormality of the eye lens (cataract) 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, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.

   These are not all the possible side effects of TRIKAFTA. Please click the product link to see the full Prescribing Information for TRIKAFTA.

   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 pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

   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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. 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, including, without limitation, statements made by Dr. Carmen Bozic in this press release, statements regarding our plans to submit an MAA in the EU and other global regulatory filings in additional markets, our expectations regarding the number of patients newly eligible for TRIKAFTA, and statements regarding the potential benefits of TRIKAFTA. 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 the sNDA to expand the use of TRIKAFTA to include children ages 6 through 11 could not be approved on a timely basis, or at all, 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, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at www.vrtx.com and on the SEC's website at www.sec.gov. 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.

   (VRTX-GEN)

   

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