VAXIMM to Participate at Upcoming Industry Events

On January 30, 2020 VAXIMM AG, a Swiss/German biotech company focused on developing oral T-cell immunotherapies, reported that the Company will participate in several industry events in the coming months (Press release, Vaximm, JAN 30, 2020, View Source [SID1234553685]). Company representatives will be available for networking and one-on-one meetings at the following conferences:

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

BIO CEO & Investor Conference
February 10-11, 2020
New York, NY, USA
VAXIMM representatives will be available for one-on-one meetings during the conference. To schedule a meeting, please use the event partnering system or contact [email protected].

13th SACHS Annual European Life Science CEO Forum
February 19-20, 2020
Zurich, Switzerland
Dr. Heinz Lubenau, CEO, will give a corporate presentation on February 19th at 2:15 pm CET and participate in the panel, "Oncology Advanced Therapies & Diagnostics," taking place on the same day at 4:30 pm. The Company will also host one-on-one meetings at this event. To request a meeting with VAXIMM, please sign up through the event’s meeting system.

14th Annual International Partnering Conference BIO-Europe Spring
March 23-25, 2020
Paris, France
VAXIMM plans to give a corporate presentation and host one-on-one meetings at this event. To request a meeting, please sign up through the event’s partneringONE meeting system.

30th MedTech Investing Europe Conference
March 31-April 1, 2020
Lausanne, Switzerland
VAXIMM representatives will be available for one-on-one meetings during the conference. Dr. Heinz Lubenau also plans to give a corporate presentation. To schedule a meeting, please contact [email protected].

01/30/2020: THERADIAG announces annual revenue of € 9.6 million in 2019, up 8.3%

On January 30, 2020 THERADIAG (ISIN: FR0004197747, Mnemonic: ALTER), a company specializing in in vitro diagnostics of autoimmune diseases and Theranostics reported its consolidated annual sales and its cash level at December 31, 2019, data under audit (Press release, Theradiag, JAN 30, 2020, View Source [SID1234553704]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

2019 consolidated revenue of € 9.6 million

As of December 31, 2019, Theradiag generated revenue of 9.6 million euros compared to 8.9 million in 2018, an increase of + 8.3% in line with the strategic axes set by the company.

The Theranostics activity recorded strong growth of + 10.5%, driven by sales of routine LISA TRACKER kits which now represent the company’s recurring activity in this segment. These kit sales exceeded a threshold with 4.3 million euros on the 2019 annual global (vs. 3.9 million at December 31, 2018). In the future, the Theranostics activity may be assimilated to this recurring activity of kit sales, now well in place, and the company will communicate on this one element of global Theranostics.

In France, where the model is now established, sales in Theranostics were particularly satisfactory with growth of 19%.

In the United States, Theradiag has concluded a year of transition reflecting the exit of its former partner in this territory. The transfer to its new partner Halio DX is gradual and will be finalized at the end of the first quarter of 2020.

In export, the company maintains a very satisfactory level of sustained activity, particularly in Spain and the United Kingdom, two strategic countries.

The In Vitro Diagnostics (IVD) activity posted overall growth in sales of + 6.0% with 5.2 million euros at the end of December 2019. It should be noted that this growth includes 1 st semester non-recurring instrumentation sales . Aside from this exceptional element, sales in this historic business segment are, as expected, slightly down. Theradiag pursues in this historical segment, in addition to its primary activity of autoimmunity, a strategy of niche specialization, such as genetics or male fertility.

Cash position

At December 31, 2019, Theradiag’s free cash flow stood at 2.9 million euros compared to 3.4 million euros at December 31, 2018 and 3.2 million euros at June 30, 2019.

Bertrand de Castelnau, CEO of Theradiag commented, "Theradiag and all the teams have made good progress in 2019: new products, new partners, new markets. We followed our roadmap with the objective of developing growth in our strategic priority markets, without compromising the investments necessary for the future of Theradiag. Priority has been given to restructuring the mix of our activities and setting milestones for internationalization, in market segments with higher profitability potential. We are staying the course to gradually improve our financial performance indicators. "

"This year is indeed a pivotal year. The overall sales growth is positive and points in the right direction. The development of the strategy around partnerships centered on Theranostics is gradually unfolding, we have recorded great successes this year and keep our commitment to innovation which is one of our key success factors. " Added Pierre Morgon, Chairman of the Board of Directors of Theradiag.

Reminder of the main events of 2019

Partnership with three new strategic distributors in Asia, December 2019
CE marking of the new i-Track 10 automated system for monitoring biotherapies, November 2019
Strategic agreement with PredictImmune for the marketing of PredictSURE IBD in French-speaking European countries, October 2019
Signing of an agreement with Halio DX as part of its development in the United States, May 2019
Signature of a development and distribution agreement with Immunodiagnostic Systems (IDS) regarding access to the latest generation IDS-i10 controller , April 2019
End of legal dispute with HOB Biotech, April 2019

Financial calendar:

2019 annual results , March 18, 2020, after market close
Annual General Meeting , May 14, 2020

Next congresses in which Theradiag participates:

12-15 February 2020 15 th congress ECCO (European Crohn’s and Colitis Organization), Vienna, Austria
5-6 March 2020: Days of the Syndicate of Young Medical Biologists – Biomed-J 2020, Paris
March 6-7, 2020: Seminar Therapeutic Study of Inflammatory Conditions of the Digestive Tube (GETAID), Paris
March 26-29, 2020: Francophone Days of Hepato-gastroenterology and Digestive Oncology 2020 (JFHOD), Paris.

AnPac Bio Announces Pricing of Initial Public Offering

On January 30, 2020 AnPac Bio-Medical Science Co., Ltd. ("AnPac Bio" or the "Company") (NASDAQ: ANPC), a biotechnology company with operations in China and the United States focused on early cancer screening and detection, reported that it has priced its initial public offering of 1,333,360 American depositary shares ("ADSs") at US$12.00 per ADS for a total offering size of approximately US$16.0 million, assuming the underwriters do not exercise their over-allotment option to purchase additional ADSs (Press release, Anpac Bio, JAN 30, 2020, View Source [SID1234553720]). Each ADS represents one Class A ordinary share of the Company. The ADSs are expected to begin trading on the Nasdaq Global Market on January 30, 2020 under the ticker symbol "ANPC."

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Company has granted the underwriters an option, exercisable within 30 days from the date of the final prospectus, to purchase up to an aggregate of 200,004 additional ADSs at the initial public offering price, less underwriting discounts and commissions, to cover over-allotments.

WestPark Capital, Inc. and Univest Securities, LLC are acting as joint bookrunners of this offering and as the representatives of the underwriters.

AnPac Bio’s registration statement relating to the offering has been filed with, and declared effective by, the United States Securities and Exchange Commission. This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities described herein, nor shall there be any sale of, these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

Thermo Fisher Scientific Reports Fourth Quarter and Full Year 2019 Results

On January 30, 2020 Thermo Fisher Scientific Inc. (NYSE: TMO), the world leader in serving science, reported its financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Thermo Fisher Scientific, JAN 30, 2020, View Source [SID1234553687]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Fourth Quarter and Full Year 2019 Highlights

•Fourth quarter revenue increased 5% to $6.83 billion.
•Fourth quarter GAAP diluted earnings per share (EPS) increased 12% to $2.49.
•Fourth quarter adjusted EPS increased 9% to $3.55.

•Full year revenue grew 5% to $25.54 billion.
•Full year GAAP diluted EPS increased 27% to $9.17.
•Full year adjusted EPS increased 11% to $12.35.

•Delivered another excellent year of high-impact innovation, launching new products across our businesses, highlighted by the Thermo Scientific Orbitrap Exploris 480 and Eclipse Tribrid mass spectrometers, Thermo Scientific Krios G4 cryo-electron microscope, Applied Biosystems QuantStudio 6 and 7 Pro real-time PCR systems, the addition of numerous allergens to the Thermo Scientific ImmunoCAP menu and, in the fourth quarter, the launch of our new Ion Torrent Genexus next-generation sequencing instrument.

•Built on our industry-leading scale in emerging and high-growth markets, opening new customer solution centers in Seoul and Shanghai for life sciences applications, new centers in Beijing and Delhi for improving food quality and safety, and a new pharma and biotech customer center in Shanghai to accelerate development of new drug therapies.

•Significantly strengthened our pharma services network in 2019 to develop and deliver high-quality medicines to patients around the world by acquiring leading viral vector manufacturer Brammer Bio and the GSK active pharmaceutical ingredient manufacturing site in Ireland, and expanding production capacity at facilities in North America and Europe to support growing demand for biologics and gene therapies.

•Continued to successfully execute our capital deployment strategy during the year, completing $1.8 billion of bolt-on acquisitions, returning $1.8 billion of capital to shareholders through stock buybacks and dividends, and refinancing $5.6 billion of debt to generate $80 million of savings annually.

Adjusted EPS, adjusted operating income, adjusted operating margin and free cash flow are non-GAAP measures that exclude certain items detailed later in this press release under the heading "Use of Non-GAAP Financial Measures."

"We finished the year strong and exceeded the goals we set out to accomplish in 2019," said Marc N. Casper, president and chief executive officer of Thermo Fisher Scientific. "From a financial perspective, we again delivered excellent revenue and earnings growth, along with strong cash flow performance.

"We continued to execute our proven growth strategy to create a differentiated experience for our customers and gain share. Across our technology-focused businesses, we extended our leading innovation track record to support our customers’ work in life sciences, clinical and applied markets. In high-growth and emerging markets, we opened new solution centers to help our customers address their challenges in a range of applications. We also strengthened our pharma services and bioproduction capabilities through a combination of strategic acquisitions and capacity investments."

Casper added, "We’re pleased to deliver another excellent year, and are even more excited about our prospects ahead as we kick off the next decade an even stronger company."

Fourth Quarter 2019

Revenue for the quarter grew 5% to $6.83 billion in 2019, versus $6.51 billion in 2018. Organic revenue growth was 5%; acquisitions, net of a divestiture, increased revenue by 1% and currency translation decreased revenue by 1%.

GAAP Earnings Results

GAAP diluted EPS in the fourth quarter of 2019 increased 12% to $2.49, versus $2.22 in the same quarter last year. GAAP operating income for the fourth quarter of 2019 grew to $1.23 billion, compared with $1.15 billion in the year-ago quarter. GAAP operating margin increased to 18.0%, compared with 17.6% in the fourth quarter of 2018.

Non-GAAP Earnings Results

Adjusted EPS in the fourth quarter of 2019 increased 9% to $3.55, versus $3.25 in the fourth quarter of 2018. Adjusted operating income for the fourth quarter of 2019 grew 5% compared with the year-ago quarter. Adjusted operating margin increased to 24.9%, compared with 24.8% in the fourth quarter of 2018.

Full Year 2019

Revenue for the full year grew 5% to $25.54 billion in 2019, versus $24.36 billion in 2018. Organic revenue growth was 6%; acquisitions, net of a divestiture, increased revenue by 1% and currency translation decreased revenue by 2%.

GAAP Earnings Results

GAAP diluted EPS for the full year increased 27% to $9.17, versus $7.24 in 2018. GAAP operating income for 2019 grew to $4.59 billion, compared with $3.78 billion a year ago. GAAP operating margin increased to 18.0% in 2019, compared with 15.5% in 2018. GAAP results for 2019 reflect the gain on the sale of the company’s Anatomical Pathology business during the second quarter.

Non-GAAP Earnings Results

Adjusted EPS for the full year rose 11% to $12.35, versus $11.12 in 2018. Adjusted operating income for 2019 grew 6% compared with 2018, and adjusted operating margin increased to 23.4%, compared with 23.1% a year ago.

Annual Guidance for 2020

The company will provide 2020 financial guidance during its earnings conference call this morning at 8:30 a.m. Eastern time.

Segment Results

Management uses adjusted operating results to monitor and evaluate performance of the company’s four business segments, as highlighted below. Since these results are used for this purpose, they are also considered to be prepared in accordance with GAAP.

Life Sciences Solutions Segment

In the fourth quarter of 2019, Life Sciences Solutions Segment revenue grew 8% to $1.84 billion, compared with revenue of $1.70 billion in the fourth quarter of 2018. Segment adjusted operating margin increased to 37.5%, versus 36.8% in the 2018 quarter.

For the full year 2019, Life Sciences Solutions Segment revenue rose 9% to $6.86 billion, compared with revenue of $6.27 billion in 2018. Segment adjusted operating margin increased to 35.7% in 2019, compared with 34.4% a year ago.

Analytical Instruments Segment

Analytical Instruments Segment revenue was $1.52 billion in the fourth quarter of 2019, compared with revenue of $1.57 billion in the fourth quarter of 2018. Segment adjusted operating margin was 26.0%, versus 26.6% in the 2018 quarter.

For the full year 2019, Analytical Instruments Segment revenue increased 1% to $5.52 billion, compared with revenue of $5.47 billion in 2018. Segment adjusted operating margin grew to 23.1%, versus 22.8% in 2018.

Specialty Diagnostics Segment

Specialty Diagnostics Segment revenue was $0.94 billion in the fourth quarter of 2019, compared with revenue of $0.95 billion in the fourth quarter of 2018, reflecting the divestiture of the Anatomical Pathology business in June 2019. Segment adjusted operating margin was 23.7%, versus 24.5% in the 2018 quarter.

For the full year 2019, Specialty Diagnostics Segment revenue was flat at $3.72 billion, compared with 2018. Segment adjusted operating margin was 25.0%, versus 2018 results of 25.6%.

Laboratory Products and Services Segment

In the fourth quarter of 2019, Laboratory Products and Services Segment revenue grew 9% to $2.83 billion, compared with revenue of $2.60 billion in the fourth quarter of 2018. Segment adjusted operating margin increased to 13.8%, versus 13.1% in the 2018 quarter.

For the full year 2019, Laboratory Products and Services Segment revenue grew 6% to $10.60 billion, compared with revenue of $10.04 billion in 2018. Segment adjusted operating margin was 12.5% in both years.

Use of Non-GAAP Financial Measures

In addition to the financial measures prepared in accordance with generally accepted accounting principles (GAAP), we use certain non-GAAP financial measures, including adjusted EPS, adjusted operating income and adjusted operating margin, which exclude certain acquisition-related costs, including charges for the sale of inventories revalued at the date of acquisition and significant transaction costs; restructuring and other costs/income; and amortization of acquisition-related intangible assets. Adjusted EPS also excludes certain other gains and losses that are either isolated or cannot be expected to occur again with any predictability, tax provisions/benefits related to the previous items, the impact of significant tax audits or events and the results of discontinued operations. We exclude the above items because they are outside of our normal operations and/or, in certain cases, are difficult to forecast accurately for future periods. We also use a non-GAAP measure, free cash flow, which is operating cash flow, excluding net capital expenditures, and also excludes operating cash flows from discontinued operations to provide a view of the continuing operations’ ability to generate cash for use in acquisitions and other investing and financing activities. We believe that the use of non-GAAP measures helps investors to gain a better understanding of our core operating results and future prospects, consistent with how management measures and forecasts the company’s performance, especially when comparing such results to previous periods or forecasts.

For example:

We exclude costs and tax effects associated with restructuring activities, such as reducing overhead and consolidating facilities. We believe that the costs related to these restructuring activities are not indicative of our normal operating costs.

We exclude certain acquisition-related costs, including charges for the sale of inventories revalued at the date of acquisition and significant transaction costs. We exclude these costs because we do not believe they are indicative of our normal operating costs.

We exclude the expense and tax effects associated with the amortization of acquisition-related intangible assets because a significant portion of the purchase price for acquisitions may be allocated to intangible assets that have lives of 3 to 20 years. Based on acquisitions closed through the end of the fourth quarter of 2019, adjusted EPS will exclude approximately $3.21 of expense for the amortization of acquisition-related intangible assets. Exclusion of the amortization expense allows comparisons of operating results that are consistent over time for both our newly acquired and long-held businesses and with both acquisitive and non-acquisitive peer companies.

We also exclude certain gains/losses and related tax effects, the impact of significant tax audits or events (such as changes in deferred taxes from enacted tax rate changes or the estimated initial impacts of U.S. tax reform legislation), which are either isolated or cannot be expected to occur again with any predictability and that we believe are not indicative of our normal operating gains and losses. For example, we exclude gains/losses from items such as the sale of a business or real estate, gains or losses on significant litigation-related matters, gains on curtailments of pension plans, the early retirement of debt and discontinued operations.

We also report free cash flow, which is operating cash flow, excluding net capital expenditures, and also excludes operating cash flows from discontinued operations to provide a view of the continuing operations’ ability to generate cash for use in acquisitions and other investing and financing activities.

Thermo Fisher’s management uses these non-GAAP measures, in addition to GAAP financial measures, as the basis for measuring the company’s core operating performance and comparing such performance to that of prior periods and to the performance of our competitors. Such measures are also used by management in their financial and operating decision-making and for compensation purposes.

The non-GAAP financial measures of Thermo Fisher’s results of operations and cash flows included in this press release are not meant to be considered superior to or a substitute for Thermo Fisher’s results of operations prepared in accordance with GAAP. Reconciliations of such non-GAAP financial measures to the most directly comparable GAAP financial measures are set forth in the accompanying tables. Thermo Fisher does not provide GAAP financial measures on a forward-looking basis because we are unable to predict with reasonable certainty, and without unreasonable effort, items such as the timing and amount of future restructuring actions and acquisition-related charges as well as gains or losses from sales of real estate and businesses, the early retirement of debt and the outcome of legal proceedings. The timing and amount of these items are uncertain and could be material to Thermo Fisher’s results computed in accordance with GAAP.

Conference Call

Thermo Fisher Scientific will hold its earnings conference call today, January 30, at 8:30 a.m. Eastern time. To listen, dial (877) 273-7122 within the U.S. or (647) 689-5496 outside the U.S. You may also listen to the call live on our website, www.thermofisher.com, by clicking on "Investors." You will find this press release, including the accompanying reconciliation of non-GAAP financial measures and related information, in that section of our website under "Financial Results." An audio archive of the call will be available under "Webcasts and Presentations" through Friday, February 7, 2020.

Vertex Reports Full-Year and Fourth-Quarter 2019 Financial Results

On January 30, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the full year and fourth quarter ended December 31, 2019 and provided full-year 2020 financial guidance (Press release, Vertex Pharmaceuticals, JAN 30, 2020, View Source [SID1234553705]).
"In 2019, our differentiated strategy of investing in serial scientific innovation to create transformative medicines delivered remarkable performance. This was marked by the early approval of TRIKAFTA, the rapid progression of our pipeline in additional diseases and continued financial growth as we continued to treat more patients with our medicines worldwide," said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. "This success is the culmination of many years of hard work to build a team, a pipeline and a company that we believe will discover, develop and commercialize transformative medicines for years to come."
Fourth-Quarter and Full-Year 2019 Financial Highlights

^Non-GAAP product revenues exclude $155.8 million related to ORKAMBI in the fourth quarter and full year ended December 31, 2019. Please refer to Note 4 for further information.
^^ GAAP Net income and GAAP Net income per share – diluted included a benefit from income taxes of approximately $1.5 billion in the fourth quarter and full year ended December 31, 2018 due to the release of Vertex’s valuation allowance on the majority of its net operating losses and other deferred tax assets.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Total GAAP and Non-GAAP product revenues increased 37% and 32%, respectively, compared to 2018, primarily driven by the global uptake of SYMDEKO and SYMKEVI in patients ages 12 and older, label expansions for the company’s CF medicines globally, and the early approval and launch of TRIKAFTA in the U.S.
GAAP net income decreased compared to 2018, largely driven by the release of Vertex’s tax valuation allowance in 2018.
Non-GAAP net income increased compared to 2018, driven by the strong growth in total product revenues.
Cash, cash equivalents and marketable securities as of December 31, 2019 were $3.8 billion, an increase of approximately $600 million compared to $3.2 billion as of December 31, 2018.

Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to 2018, 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 new disease areas. Combined GAAP R&D and SG&A expenses also increased compared to 2018 due to Vertex’s business development activities.

GAAP and Non-GAAP income taxes increased significantly compared to 2018 due to Vertex’s release of its valuation allowance on the majority of its deferred tax assets in the fourth quarter of 2018. GAAP and non-GAAP income taxes in 2019 include a provision for income taxes on Vertex’s pre-tax income using an estimated effective tax rate approximating statutory rates. This provision for income taxes includes a significant non-cash charge due to Vertex’s ability to offset its pre-tax income against previously benefited net operating losses. Refer to "Supplemental Income Tax Information" for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.

Total GAAP and Non-GAAP product revenues increased 63% and 45%, respectively, compared to the fourth quarter of 2018, primarily driven by the early approval and launch of TRIKAFTA in the U.S. and the global uptake of SYMDEKO and SYMKEVI in patients ages 12 and older.
GAAP net income decreased compared to the fourth quarter of 2018, largely driven by the release of Vertex’s tax valuation allowance in the fourth quarter of 2018.
Non-GAAP net income increased compared to the fourth quarter of 2018, driven by the strong growth in total product revenues.

Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to the fourth quarter of 2018, 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 new disease areas.
GAAP and Non-GAAP income taxes increased significantly compared to the fourth quarter of 2018 due to Vertex’s release of its tax valuation allowance in the fourth quarter of 2018. Refer to "Full-Year 2019 Expenses" for further information. Refer to "Supplemental Income Tax Information" for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.

Full-Year 2020 Financial Guidance
Vertex today provided its full-year 2020 financial guidance. A summary of the company’s current financial expectations is below:

"Entering 2020, Vertex has never been stronger," said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer. "We are well-positioned for both near- and long-term growth based on treating more people with our CF medicines and from other future medicines in diseases aligned with our strategy, including alpha-1 antitrypsin deficiency, APOL1-mediated kidney diseases, pain and severe hemoglobinopathies. Going forward, our financial strength will enable us to continue to significantly invest

in internal R&D and external innovation, which will provide access to new technologies, programs and expertise that will lead to further growth in the years ahead."

Clinical Development
Cystic Fibrosis (CF):

On October 21, 2019, the company announced that the U.S. Food and Drug Administration (FDA) approved TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of CF in people ages 12 years and older who have at least one F508del mutation. TRIKAFTA is Vertex’s fourth breakthrough medicine approved to treat the underlying cause of CF.

On October 31, 2019, the company announced that the European Medicines Agency (EMA) has validated the marketing authorization application (MAA) for the elexacaftor, tezacaftor and ivacaftor triple combination regimen.

Enrollment is ongoing in a Phase 3 study evaluating the elexacaftor, tezacaftor and ivacaftor triple combination regimen in children with CF ages 6 to 11 years who have two F508del mutations and in children who have one F508del mutation and one minimal function mutation. Pending the results of the Phase 3 study, the company plans to submit a supplemental New Drug Application (sNDA) to the FDA in 2020 followed by additional submissions to other global regulatory agencies.
Alpha-1 Antitrypsin (AAT) Program:

VX-814: Enrollment is ongoing in the Phase 2 proof-of-concept study evaluating VX-814, the company’s first oral small molecule corrector for the treatment of alpha-1 antitrypsin (AAT) deficiency. Vertex expects to obtain data from this study in 2020.

VX-864: A Phase 1 study is ongoing in healthy volunteers evaluating VX-864, the company’s second investigational small molecule corrector for the treatment of AAT deficiency.
Sickle Cell Disease and Beta Thalassemia:

In November 2019, Vertex and its partner CRISPR Therapeutics announced positive, interim data from the first two patients with severe hemoglobinopathies – one patient with transfusion-dependent beta thalassemia and one patient with severe sickle cell disease – treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in the ongoing Phase 1/2 clinical trials. Enrollment is ongoing in both trials and the companies expect to provide additional data for this program in 2020.

APOL1-Mediated Kidney Diseases:

Vertex continues to advance a portfolio of molecules for the treatment of APOL1-mediated focal segmental glomerulosclerosis (FSGS) and other serious kidney diseases. In the fourth quarter of 2019, Vertex completed a Phase 1 study of its first investigational oral small molecule VX-147 in healthy volunteers. Vertex plans to initiate a Phase 2 proof-of-concept study in 2020 to evaluate the reduction in protein levels with VX-147.
Pain:

The company announced today that it has discontinued Phase 1 development of VX-961. The company continues to advance a portfolio of NaV1.8 inhibitors into the clinic. As part of this strategy, Vertex plans to advance an additional molecule into clinical development in the first half of 2020.
Type 1 Diabetes:

Vertex plans to advance its cell therapy program for the treatment of type 1 diabetes into clinical development in late 2020 or early 2021.

Notes and Explanations
1: The company records gains and losses related to changes in the fair value of its strategic investments to "Other income, net."
2: In the fourth quarter of 2018, the company recorded a non-cash benefit from income taxes of approximately $1.5 billion related to the release of its valuation allowance on the majority of its net operating losses and other deferred tax assets. As a result, the company recorded deferred tax assets of $1.5 billion on its consolidated balance sheet as of December 31, 2018, which were previously subject to its valuation allowance. Starting in the first quarter of 2019, the company began recording a provision for income taxes on its pre-tax income using an estimated effective tax rate that approximates statutory rates. The provision includes a significant non-cash charge due to the company’s ability to offset its pre-tax income against previously benefited net operating losses. The company expects the majority of its tax provision to represent a non-cash expense until its net operating losses have been fully utilized. As of December 31, 2019, the company’s federal net operating losses and credits that were available to offset future pre-tax income were approximately $3.5 billion.
3: During the three and twelve months ended December 31, 2019, the increase in the fair value of the contingent consideration relates to payments potentially payable to Exonics’ former equity holders. During the three and twelve months ended December 31, 2018, the company consolidated the financial statements of a variable interest entity, or VIE, resulting in changes in the fair value of contingent consideration.
4: "ORKAMBI adjustment" in the three and twelve months ended December 31, 2019 includes 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 and cost of sales, which related to prior period shipments of ORKAMBI distributed through the early access program in France. The company has 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. Beginning in 2020, the company does not expect to adjust any revenues from its GAAP net product revenues.

5: "Collaborative revenues and expenses" in the three and twelve months ended December 31, 2019 and 2018 primarily related to collaborative upfront and milestone payments. "Collaborative revenues and expenses" in the three and twelve months ended December 31, 2018 also included revenues and expenses attributable to our VIE’s operations.
6: "Acquisition-related costs" in the three and twelve months ended December 31, 2019 primarily related to costs associated with the company’s acquisitions of Semma and Exonics. There were no comparable amounts during the three and twelve months ended December 31, 2018.
7: In the three and twelve months ended December 31, 2019, "Estimated income taxes related to non-GAAP adjustments to pre-tax income" primarily related to (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 upfront payments. In the three and twelve months ended December 31, 2018, "Estimated income taxes related to non-GAAP adjustments to pre-tax income" were related to a provision for income taxes attributable to the company’s VIE and excess tax benefits related to stock-based compensation.