On February 27, 2019 Acceleron Pharma Inc. (Nasdaq: XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Acceleron Pharma, FEB 27, 2019, View Source [SID1234533804]).

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"A series of significant achievements in 2018 have positioned Acceleron to execute on a number of key milestones across the entire pipeline during 2019 and 2020," said Habib Dable, President and Chief Executive Officer of Acceleron. "First and foremost, we and our global collaboration partner, Celgene, plan to submit marketing applications in the U.S. and E.U. for our lead product candidate, luspatercept, in lower-risk myelodysplastic syndromes and transfusion-dependent beta-thalassemia in the first half of the year. At the same time, we’re continuing to evaluate luspatercept’s potential to treat a range of anemias, from first-line therapy in MDS via the ongoing COMMANDS Phase 3 trial, to non-transfusion-dependent beta-thalassemia, myelofibrosis and beyond."

"In addition, our wholly-owned programs in neuromuscular and pulmonary disease are all advancing. We’re anticipating topline results from the placebo controlled part of our Phase 2 trials evaluating our locally-acting muscle agent, ACE-083, in FSHD and CMT by the end of the year. Lastly, 2020 will bring important Phase 2 trial results in PAH with sotatercept, which we believe has the potential to alter the treatment landscape for this devastating disease."

Development Program Highlights

Hematology

Luspatercept: Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is an investigational first-in-class erythroid maturation agent (EMA) designed to address a late-stage erythroid maturation defect that results in chronic anemia and the need for regular red blood cell transfusions in adults with serious hematologic diseases. Luspatercept is part of the global collaboration between Acceleron and Celgene.

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The MEDALIST and BELIEVE Phase 3 trial results in patients with lower-risk MDS and transfusion-dependent beta-thalassemia, respectively, were presented at the 60th ASH (Free ASH Whitepaper) Annual Meeting and Exposition in December 2018.

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The MEDALIST and BELIEVE presentations were both selected for presentation during the "Best of ASH (Free ASH Whitepaper)" session at the meeting.

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Acceleron and Celgene plan to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for luspatercept in patients with anemia related lower-risk MDS and beta-thalassemia in April 2019.

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Acceleron and Celgene remain on track to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for luspatercept in patients with anemia related to lower-risk MDS and beta-thalassemia in the first half of 2019.

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The ongoing Phase 2 trial of luspatercept in patients with MF has completed target enrollment, with preliminary results expected in the second half of 2019.

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Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS and the BEYOND Phase 2 trial in patients with non-transfusion-dependent beta-thalassemia, with preliminary results expected from the BEYOND trial in 2020.

Neuromuscular Disease

ACE-083: Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie-Tooth Disease (CMT)
ACE-083 is an investigational locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause focal muscle weakness. ACE-083 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta superfamily ligands involved in muscle formation.

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Preliminary results from Part 1 of the Phase 2 trials evaluating ACE-083 in patients with FSHD and CMT, were presented at the 2018 World Muscle Society (WMS) Annual Meeting in October 2018.

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Part 2 of the Phase 2 FSHD trial has completed patient enrollment, with preliminary topline results expected in the second half of 2019.

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Enrollment is ongoing in Part 2 of the Phase 2 CMT trial, with preliminary results expected by the end of 2019.

ACE-2494: Neuromuscular Disease
ACE-2494 is designed to have a systemic effect on muscle mass and strength for diseases that cause muscle weakness throughout the body. ACE-2494 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta superfamily ligands involved in muscle formation.

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Enrollment is ongoing in the Phase 1 healthy volunteer trial, with preliminary results expected in the first half of 2019.

Pulmonary Disease

Sotatercept: Pulmonary Arterial Hypertension (PAH)
Sotatercept acts as a multi-ligand trap for certain members of the TGF-beta superfamily to rebalance BMPRII signaling, which is a key molecular driver of PAH. In preclinical studies of PAH, sotatercept reversed pulmonary vessel muscularization and improved indicators of right heart failure.

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Preclinical results from multiple studies of sotatercept in PAH were presented at the American Heart Association Scientific Sessions in November 2018.

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Enrollment is ongoing in the PULSAR Phase 2 trial in patients with PAH, with preliminary results expected in the first half of 2020.

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The exploratory SPECTRA trial in patients with PAH has been initiated, with preliminary results expected in 2020.

Corporate Highlights

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The Company recently raised approximately $264.5 million of gross proceeds in a follow-on offering of common stock.

Financial Results
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Cash Position – Cash, cash equivalents and investments as of December 31, 2018 were $291.3 million. As of December 31, 2017, the Company had cash, cash equivalents and investments of $372.9 million. Based on the Company’s current operating plan and projections, it believes that current cash, cash equivalents and investments, together with the net proceeds of $248.2 million from its recent

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common stock offering, will be sufficient to fund projected operating requirements until such time as it expects to receive significant royalty revenue from luspatercept sales.
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Revenue – Collaboration revenue for the year was $14.0 million. The revenue is all from the Company’s partnership with Celgene and is primarily related to expenses incurred by the Company in support of luspatercept.
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Costs and Expenses – Total costs and expenses for the year were $138.4 million. This includes R&D expenses of $103.9 million and G&A expenses of $34.5 million.
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Net Loss – The Company’s net loss for the year ended December 31, 2018 was $118.9 million.

Conference Call and Webcast
The Company will host a webcast and conference call to discuss its fourth quarter and full year financial results for 2018 and provide an update on recent corporate activities on February 27, 2019, at 5:00 p.m. EST.

The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron Fourth Quarter and Full Year 2018 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On February 27, 2019 Context Therapeutics, a clinical-stage biotechnology company, reported that new preclinical data on CTX-30916, a new PR antagonist, will be featured at the upcoming AACR (Free AACR Whitepaper) Annual Meeting taking place in Atlanta, Georgia (Press release, Context Therapeutics, FEB 27, 2019, View Source [SID1234533830]). The data will be presented during the Receptors and Growth Factors session on April 1, 2019 from 1:00 PM – 5:00 PM.

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At the conference Dr. Deepak Lala, Ph.D., Chief Technology Officer for Context Therapeutics and Dr. Tasir Haque Ph.D., Head of Chemistry for Context Therapeutics will present preclinical data highlighting the biochemical and cellular activity of CTX-30916 including its mechanism of action in inhibiting breast cancer cell proliferation. CTX-30916 is a next generation PR antagonist with potential for the treatment of breast, uterine, and ovarian cancers.

The poster details are as follows:

Poster Title: Identification of CTX-30916 as a new antagonist of progesterone receptor signaling pathways

Session Category: Molecular and Cellular Biology / Genetics

Session Title: Receptors and Growth Factors

Session Date and Time: Monday Apr 1, 2019 1:00 PM – 5:00 PM

Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 38

Poster Board Number: 4

Abstract Number: 2625

For more information on AACR (Free AACR Whitepaper) 2019, please visit View Source

On February 27, 2019 PRA Health Sciences, Inc. ("PRA" or the "Company") (NASDAQ: PRAH) reported financial results for the quarter and year ended December 31, 2018 (Press release, PRA Health Sciences, FEB 27, 2019, View Source;p=irol-newsArticle&ID=2389364 [SID1234533741])

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"We are delighted to have closed out 2018 with another quarter of strong financial results," said Colin Shannon, PRA’s Chief Executive Officer. "Our ability to execute across all of our businesses has allowed us to deliver solid revenue growth and strong bottom-line results. Our key financial metrics continue to improve, as highlighted by our 1.30 net book-to-bill ratio and our expanding margins. We remain focused on providing broad and flexible services to our clients and believe we are well positioned to deliver strong results in 2019."

Net new business for our Clinical Research segment for the three months ended December 31, 2018 was $667.3 million, representing a net book-to-bill ratio of 1.30 for the period. This net new business contributed to an ending backlog of $4.2 billion at December 31, 2018.

For the three months ended December 31, 2018, revenue was $729.6 million, which represents growth of 11.2%, or $73.8 million, compared to the fourth quarter of 2017 at actual foreign exchange rates. On a constant currency basis, revenue grew $79.0 million, an increase of 12.0% compared to the fourth quarter of 2017. By segment, the Clinical Research segment generated revenues of $655.6 million, while the Data Solutions segment generated revenues of $74.0 million.

On January 1, 2018, the Company adopted Accounting Standards Codification Topic 606, "Revenue from Contracts with Customers," or ASC 606, using the modified retrospective method for all contracts that were not completed as of January 1, 2018. Prior periods have not been restated under this guidance and remain as previously reported. The primary impact of applying this new guidance on our statement of operations is that (i) we now recognize reimbursements from our customers for payments to investigators as revenue, whereas these payments and costs were previously recorded on a net basis, and (ii) we include all reimbursed costs in the total project costs when measuring our progress under our research contracts instead of recording these amounts on a separate basis.

Excluding the impact of the adoption of ASC 606 and reimbursement revenue, revenue increased $20.2 million, which represents growth of 3.6% at actual foreign exchange rates and 4.2% on a constant currency basis.

Direct costs, exclusive of depreciation and amortization, were $365.7 million during the three months ended December 31, 2018 compared to $368.9 million for the three months ended December 31, 2017. The decrease in direct costs was primarily due to a favorable impact of $8.6 million from foreign currency exchange rate fluctuations, which was offset by an increase in salaries and related benefits of $3.6 million in our Clinical Research segment as we continue to ensure appropriate staffing levels and an increase of $1.6 million in our Data Solutions segment. Direct costs were 50.1% of revenue during the fourth quarter of 2018 compared to 56.2% of revenue during the fourth quarter of 2017. Excluding the impact of the adoption of ASC 606 and reimbursement revenue, direct costs were 62.1% of revenue during the fourth quarter of 2018 compared to 64.9% of revenue during the fourth quarter of 2017.

Selling, general and administrative expenses were $96.4 million during the three months ended December 31, 2018 compared to $92.2 million for the three months ended December 31, 2017. Excluding the impact of the adoption of ASC 606 and reimbursement revenue, selling, general and administrative costs were 16.4% of revenue during the fourth quarter of 2018 compared to 16.2% of revenue during the fourth quarter of 2017.

GAAP net income was $71.5 million for the three months ended December 31, 2018, or $1.07 per share on a diluted basis, compared to a GAAP net loss of $16.0 million for the three months ended December 31, 2017, or $0.25 per share on a diluted basis. Our reported net loss for the three months ended December 31, 2017 included the loss on modification of debt and the revaluation of acquisition-related earn-out liabilities.

EBITDA was $124.9 million for the three months ended December 31, 2018, representing an increase of 720.9% compared to the three months ended December 31, 2017. Our EBITDA for the three months ended December 31, 2017 included the loss on modification of debt and the revaluation of acquisition-related earn-out liabilities. Adjusted EBITDA was $136.2 million for the three months ended December 31, 2018, representing growth of 18.8% compared to the three months ended December 31, 2017.

Adjusted net income was $86.9 million for the three months ended December 31, 2018, representing 26.4% growth compared to the three months ended December 31, 2017. Adjusted net income per diluted share was $1.31 for the three months ended December 31, 2018, representing 26.0% growth compared to the three months ended December 31, 2017.

Full Year 2018 Financial Highlights

For the twelve months ended December 31, 2018, revenue was $2,871.9 million, which represents growth of 27.1%, or $612.5 million, compared to the twelve months ended December 31, 2017 at actual foreign exchange rates. On a constant currency basis, revenue grew $597.3 million, representing growth of 26.4% compared to the twelve months ended December 31, 2017. By segment, the Clinical Research segment generated revenues of $2,622.4 million, while the Data Solutions segment generated revenues of $249.5 million.

Excluding the impact of the adoption of ASC 606 and reimbursement revenue, revenue increased $348.5 million, which represents growth of 17.9% at actual foreign exchange rates and 17.4% on a constant currency basis. Organic revenue growth, excluding the adoption of ASC 606, reimbursement revenue and revenue attributable to our Data Solutions segment, was 10.2% at actual foreign exchange rates and 9.7% on a constant currency basis.

Reported GAAP income from operations was $281.3 million, reported GAAP net income attributable to PRA Health Sciences was $153.9 million and reported GAAP net income attributable to PRA Health Sciences per diluted share was $2.32 for the twelve months ended December 31, 2018.

Adjusted net income was $284.1 million for the twelve months ended December 31, 2018, an improvement of 29.8% compared to the twelve months ended December 31, 2017. Adjusted net income per diluted share was $4.28 for the twelve months ended December 31, 2018, up 28.5% compared to the twelve months ended December 31, 2017.

Full Year 2019 and Q1 2019 Guidance

For full year 2019, the Company expects to achieve total revenues between $3.09 billion and $3.20 billion, representing as reported and constant currency growth of 8% to 11%. On an ASC 605 basis, the Company expects to achieve revenues of between $2.475 billion and $2.57 billion, representing as reported and constant currency growth of 8% to 12%.

We expect GAAP net income per diluted share of between $3.65 and $3.80 per share and adjusted net income per diluted share of between $4.93 and $5.08 per share, representing growth of 15% to 19%. We anticipate an annual effective income tax rate estimate of 24%.

Our effective tax rate may differ from this estimate, due to, among other things, changes to estimates of the geographic allocation of our pre-tax income as well as changes in interpretations, analysis, and additional guidance that may be issued by regulatory agencies as it relates to the Tax Cuts and Jobs Act.

For Q1 2019, the Company expects to achieve total revenues between $720.0 million and $740.0 million, representing as reported growth of 3% to 5% and constant currency growth of 4% to 7%. On an ASC 605 basis, the Company expects to achieve revenues of between $575.0 million and $595.0 million, representing as reported growth of 3% to 6% and constant currency growth of 4% to 7%. The Company expects GAAP net income per diluted share of between $0.74 and $0.79 per share, adjusted net income per diluted share between $1.05 and $1.10 per share, and an annual effective income tax rate of 24%.

Our 2019 guidance assumes a EURO rate of 1.15 and a GBP rate of 1.35 with all other foreign currencies using a rate as of January 31, 2019.

A reconciliation of our non-GAAP measures, including revenue reported on an ASC 605 basis, EBITDA, adjusted EBITDA, adjusted net income, adjusted net income per share and our 2019 guidance, to the corresponding GAAP measures is included in this press release.

Conference Call Details

PRA will host a conference call at 9:00 a.m. ET on February 28, 2019, to discuss the contents of this release and other relevant topics. To participate, please dial (877) 930-8062 within the United States or (253) 336-7647 outside the United States approximately 10 minutes before the scheduled start of the call. The conference ID for the call is 8697447. The conference call will also be accessible, live via audio broadcast, on the Investor Relations section of the PRA website at investors.prahs.com. A replay of the conference call will be available online at investors.prahs.com. In addition, an audio replay of the call will be available for one week following the call and can be accessed by dialing (855) 859-2056 within the United States or (404) 537-3406 outside the United States. The replay ID is 8697447.

Additional Information

A financial supplement with fourth quarter 2018 results, which should be read in conjunction with this press release, may be found in the Investor Relations section of our website at investors.prahs.com in a document titled "Q4 2018 Earnings Presentation."

On February 27, 2019 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq:SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported two oral presentations at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting being held March 29 – April 3, 2019 in Atlanta, Georgia (Press release, Syndax, FEB 27, 2019, View Source [SID1234533761]).

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Oral Presentation Details

Title: Identification of gene signatures associated with response in a Phase 2 trial of entinostat (ENT) plus pembrolizumab (PEMBRO) in non-small cell lung cancer (NSCLC) patients whose disease has progressed on or after anti-PD-(L)1 therapy
First author: Peter Ordentlich, Ph.D.
Session: Advances in Novel Immunotherapeutics (Clinical Trials Minisymposium)
Abstract Number: 7822
Location: Georgia World Congress Center, Building A, Room 411
Date and Time: Sunday, March 31, 2019; 3:00 p.m. – 5:00 p.m. ET

Title: Efficacy and safety of entinostat (ENT) and pembrolizumab (PEMBRO) in patients with melanoma previously treated with anti-PD1 therapy
First author: Ryan Sullivan, M.D.
Session: Optimizing PD-1/PD-L1 Immune Checkpoint Inhibitor Therapy (Clinical Trials Plenary Session)
Abstract Number: 7449
Location: Georgia World Congress Center, Building A, Marcus Auditorium
Date and Time: Monday, April 1, 2019; 10:30 a.m. – 12:30 p.m. ET

All accepted abstracts will be published in the 2019 Proceedings of the AACR (Free AACR Whitepaper). Session information is available online via the Annual Meeting Itinerary Planner through the AACR (Free AACR Whitepaper) website at www.aacr.org.

On February 27, 2019 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported financial results for the fourth quarter and year ended December 31, 2018 and provided a business update (Press release, Adaptimmune, FEB 27, 2019, View Source [SID1234533726]).

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"2018 was a year of strong delivery across the portfolio with record numbers of patients treated in our clinical trials. We moved through the dose escalation portion of our studies with ADP-A2M4 and ADP-A2M10 and we are now treating patients in the expansion phases for both programs. Our third program, ADP-A2AFP, moved to the second dose cohort at target doses of 1 billion cells. We are very pleased to have observed an acceptable safety profile, thus far, with all three programs, showing no evidence of off-target toxicity or alloreactivity," said James Noble, Chief Executive Officer. "We are now able to devote our resources to these programs following the successful transition of NY-ESO to GSK."

"We made equally impressive progress in manufacturing with our in-house facility going from our first ever dose, at the beginning of 2018, to being able to produce target doses for up to 10 patients per month. In the UK, we started up our vector manufacturing that should begin to produce vector later this year. 2019 promises to be a significant year, with data emerging across our portfolio from May onwards. We look forward to reporting clinical data throughout the year," added James Noble.

Clinical momentum

Initial safety testing is complete in the triple tumor and non-small cell lung cancer (NSCLC) studies with ADP-A2M10, as well as the basket study with ADP-A2M4. All three studies are enrolling and treating patients with up to 10 billion cells in the expansion phases with no pre-determined stagger between dosing required.

In 2018, the Safety Review Committee (SRC) endorsed dose escalation through Cohorts 1, 2, and 3 of these studies, after reviewing safety data from 16 patients treated in the ADP-A2M10 studies and nine patients in the ADP-A2M4 study.

In the hepatocellular carcinoma study with ADP-A2AFP, the SRC endorsed escalation to safety Cohort 2 to treat patients with a target dose of 1 billion cells.

To date, across all four studies, most adverse events have been consistent with those typically experienced by cancer patients undergoing cytotoxic chemotherapy or other cancer immunotherapies with no evidence of alloreactivity or toxicity related to off-target binding.

Building an integrated company

Since the opening of the Navy Yard facility in January 2018, Adaptimmune can now manufacture cells for up to 10 patients per month, and this is scalable to 100 patients per month without significant capital expenditure. Further, an additional 10 patients per month can be treated with cells produced at a third-party vendor HCATs. Both the Navy Yard and HCATs facilities are now able to routinely produce cells to meet target doses across a broad range of solid tumors. This capacity will allow Adaptimmune to service its existing and planned clinical trials.

With respect to vector, Adaptimmune is well supplied as its own vector manufacturing has completed its first engineering run with first production anticipated in 2019, as well as production in place at a third-party vendor. The vendor has already produced vector for ADP-A2M10, ADP-A2M4, ADP-A2AFP, and next generation SPEAR T-cells.

In light of the Company’s increased clinical focus, Rafael Amado assumed the new role of President of R&D last year to bring the clinical and research functions under a single leadership, facilitating alignment and integration of all parts of the R&D structure.

The Company is working with more than 20 active clinical trial sites at leading cancer centers in the US, Canada, and the EU. In Europe, the infrastructure has been tested and has delivered the first doses to patients with additional patients being enrolled in the UK and Spain. Adaptimmune has also reached agreement on an expanded collaboration with MD Anderson Cancer Center (Houston, TX) to further enhance the Company’s translational research capabilities.

While progressing studies with ADP-A2M10, ADP-A2M4, and ADP-A2AFP as well as planning for the next stage of clinical development, including potential registration trials, Adaptimmune continues to progress developing next generation SPEAR T-cells, new targets, other HLAs, and an off-the-shelf product.

Finally, Adaptimmune is funded through to late 2020, based on management’s current estimates, with Total Liquidity(1) of $205 million (including cash and cash equivalents of $68 million) at year-end 2018.

2018 Highlights

Clinical progress with wholly-owned programs:

·ADP-A2M10 (MAGE-A10):

·January 2018: The SRC endorsed escalation to Cohort 2 (1 billion target dose) in the ADP-A2M10 triple tumor and lung studies based on favorable safety data

·ASCO: Poster presented at ASCO (Free ASCO Whitepaper) with initial safety data

·July: SRC endorsed dose escalation to Cohort 3 in the NSCLC and triple tumor studies, and treating patients at target dose of 5 billion cells commenced

·ESMO: Presentation of Cohorts 1 and 2 safety data showing dose proportionate persistence and expansion

·Q4: Escalation to expansion phase allowing for doses up to 10 billion cells with no pre-determined stagger between patients for both studies

·ADP-A2M4 (MAGE-A4) — Basket study:

·ASCO: The SRC endorsed escalation to Cohort 2 based on favorable safety data in Cohort1. Added synovial sarcoma and myxoid/round cell liposarcoma (MRCLS) indications for a total of nine solid tumors in this study

·August: Dose escalation to Cohort 3

(1) Total liquidity is a non-GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.

· ESMO (Free ESMO Whitepaper): Presentation of Cohorts 1 and 2 safety data showing dose appropriate persistence and expansion, and early, but transient, evidence of antitumor activity in one ovarian cancer patient

· Q4: Escalation to expansion phase allowing for doses up to 10 billion cells with no pre-determined stagger between patients

·ADP-A2AFP (AFP) – Hepatocellular carcinoma:

· SRC endorsed escalation to Cohort 2 with target doses of 1 billion cells

· Clinical learnings

· AACR (Free AACR Whitepaper) 2018: Presented two posters with ADP-A2M10 and ADP-A2M4 preclinical data

· Q2: Published study in Cancer Discovery indicating that NY-ESO SPEAR T-cells are long-lived, self-renewing, and capable of persistent anti-tumor effects

· SITC (Free SITC Whitepaper): Updated data supporting further understanding of systemic and local immunity following NY-ESO treatment in synovial sarcoma, including the positive impact of a more intense pre-conditioning regimen with respect to SPEAR T-cell expansion and persistence; adjusted to a more intense pre-conditioning regimen in current trials as a result

· Q4: Published paper in Hepatology "Tuning T-cell receptor affinity to optimize clinical risk-benefit when targeting α-fetoprotein (AFP) positive liver cancer," which details the development of the SPEAR T-cells targeting AFP

Preclinical:

· Good progress with off-the-shelf product and presented progress to date at ASGCT (Free ASGCT Whitepaper) 2018

· Developing multiple next generation approaches — first candidate ready for IND submission in 2H 2019

·Investigated new targets and additional HLAs to be brought to the clinic beyond 2019

Well-developed preclinical package including proprietary methods for predicting binding of a TCR to an off-target peptide utilizing alanine scanning processes. A licensing program is available to third parties wishing to use this patented method.

Manufacturing:

· Routinely producing cell product at target doses across a broad range of solid tumor indications

· Navy Yard facility now capable of manufacturing cell product for up to 10 patients per month (scalable to 100 patients per year) with capacity for an additional 10 patients per month at a third-party supplier HCATs

· Implemented rapid sterility testing to decrease vein-to-vein time

· Routinely producing more than 5 billion cells to meet target doses

· Agreement with third-party vector manufacturer for commercial supply — batches manufactured for ADP-A2M10, ADP-A2M4, ADP-A2AFP, and next generation SPEAR T-cells

· In-house suspension vector manufacturing capacity with an engineering run completed and first production expected in 2019

NY-ESO Program (now transitioned to GSK):

· Myxoid/round cell liposarcoma: Initial responses observed in a second solid tumor indication with data presented at ASCO (Free ASCO Whitepaper) 2018 and updated at SITC (Free SITC Whitepaper) 2018

· Agreement with GSK:

· Completed transition of the NY-ESO SPEAR T-cell development program in August 2018

· GSK assumed full responsibility for future research, development, and potential commercialization of NY-ESO now called GSK3377794 (GSK ‘794)

· Adaptimmune received ~ $27.5 million (~£21.2 million) in 2018 from GSK for completion of the transition

Other corporate news:

·Completed Registered Direct Offering, raising total net proceeds of ~$100 million in September 2018

· Appointed John Furey as an independent Non-Executive Director, effective July 5, 2018, succeeding Dr. Peter Thompson

Financial Results for the fourth quarter and year ended December 31, 2018

·Cash / liquidity position: As of December 31, 2018, Adaptimmune had cash and cash equivalents of $68.4 million and Total Liquidity(1) of $205.1 million.

·Revenue: Revenue represents the upfront and milestone payments, which are recognized as delivered services to GSK. Revenue for the fourth quarter and year ended December 31, 2018 were $1.5 million and $59.5 million compared to $4.3 million and $37.8 million for the same periods of 2017. Revenue in the year ended December 31, 2018 includes $39.1 million of revenue for the license to NY-ESO, which commenced in September 2018.

·Research and development ("R&D") expenses: R&D expenses for the fourth quarter and year ended December 31, 2018 were $22.8 million and $98.3 million, compared to $25.1 million and $87.4 million for the same periods of 2017. The increase was primarily due to increased costs associated with clinical trials; costs of developing manufacturing capability in the Company’s U.S. facility and increased personnel expenses.

· General and administrative ("G&A") expenses: G&A expenses for the fourth quarter and year ended December 31, 2018 were $10.8 million and $43.6 million, compared $8.8 million and $31.1 million for the same periods of 2017. The increase was primarily due to increased personnel costs consistent with our planned growth an increase in costs associated with supporting and maintaining our IT infrastructure.

·Net loss: Net loss attributable to holders of the Company’s ordinary shares for the fourth quarter and year ended December 31, 2018 were $36.2 million and $95.5 million ($(0.16) per ordinary share) compared to $27.3 million and $70.1 million ($(0.13) per ordinary share) in the same periods of 2017.

Financial Guidance

The Company believes that its existing cash and cash equivalents, short-term deposits and marketable securities, Total Liquidity, will fund the Company’s current operating plan through to late 2020.

Conference Call Information

The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EST (1:00 p.m. GMT) today, February 27, 2019. The live webcast of the conference call will be available via the events page of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available after the call at the same address. To participate in the live conference call, if preferred, please dial (833) 652-5917 (U.S. or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (9455267).