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Horizon Therapeutics plc Reports Fourth-Quarter and Full-Year 2019 Financial Results; Announces Full-Year 2020 Guidance

On February 26, 2020 Horizon Therapeutics plc (Nasdaq: HZNP) reported its fourth-quarter and full-year 2019 financial results and provided its full-year 2020 net sales and adjusted EBITDA guidance (Press release, Horizon Pharma, FEB 26, 2020, View Source [SID1234554807]).

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"The fourth quarter capped off another year of tremendous progress at Horizon, marked by the achievement of several important milestones," said Timothy Walbert, chairman, president and chief executive officer, Horizon. "We are in our strongest position ever as a company, entering 2020 with FDA approval of TEPEZZA, the first and only medicine approved for the treatment of thyroid eye disease. We continue to see strong growth for KRYSTEXXA, the only approved medicine for uncontrolled gout, particularly following the significantly higher complete response rate demonstrated when used in combination with methotrexate. With the excellent growth potential we see for both TEPEZZA and KRYSTEXXA, we recently increased our peak U.S. net sales expectations to more than $1 billion for each medicine. We remain focused on optimizing the benefits our medicines provide patients and driving value for our shareholders."

TEPEZZA Approved by FDA for the Treatment of Thyroid Eye Disease (TED): On Jan. 21, 2020, the U.S. Food and Drug Administration (FDA) approved TEPEZZA (teprotumumab-trbw) for the treatment of TED, well in advance of the Prescription Drug User Fee Act (PDUFA) action date of March 8, 2020. TEPEZZA is the first and only FDA-approved medicine for the treatment of TED, a serious, progressive and vision-threatening rare autoimmune disease.

The New England Journal of Medicine Published TEPEZZA’s Phase 3 Clinical Trial Data: In January 2020, the Company announced that The New England Journal of Medicine had published the comprehensive results of Phase 3 clinical trial evaluating TEPEZZA for the treatment of TED. The results from the clinical trial demonstrated that TEPEZZA provides significant improvements in proptosis (eye bulging) and diplopia (double vision) when compared to a placebo. This is one of the few clinical programs to have both its Phase 2 and Phase 3 clinical results published in The New England Journal of Medicine.

FDA Advisory Committee Voted Unanimously to Support the Use of TEPEZZA for TED: On Dec. 13, 2019, the Dermatologic and Ophthalmic Drug Advisory Committee (DODAC) of the FDA voted unanimously (12-0) that the potential benefits of TEPEZZA outweigh the potential risks for the treatment of TED.

KRYSTEXXA MIRROR Open-Label Immunomodulation Trial Demonstrated 79 Percent Complete Response Rate: In January 2020, the Company announced topline results from its MIRROR open-label trial, which evaluated the use of the immunomodulator methotrexate with KRYSTEXXA to increase the complete response rate of KRYSTEXXA. The results of the trial demonstrated that 79 percent, or 11 of 14 patients enrolled, achieved a complete response, defined as the proportion of serum uric acid (sUA) responders (sUA <6 mg/dL) at Month 6. The 79 percent response rate is nearly double the 42 percent response rate in the KRYSTEXXA Phase 3 clinical program, which evaluated KRYSTEXXA alone. The combination was also well tolerated.

Increased Peak U.S. Annual Net Sales Expectations for Key Growth Drivers: In January 2020, the Company announced that it increased both KRYSTEXXA and TEPEZZA peak U.S. annual net sales expectations to more than $1 billion each, from the previous expectation of more than $750 million each.

Initiated PROTECT Trial Evaluating KRYSTEXXA to Improve Management of Uncontrolled Gout for Adults with a Kidney Transplant: In October 2019, the Company initiated its open-label PROTECT clinical trial evaluating the use of KRYSTEXXA in adults with uncontrolled gout who have undergone a kidney transplant. The objective of the trial is to demonstrate that KRYSTEXXA can provide effective disease control without burdening the kidneys. The randomized multicenter open-label trial is expected to enroll 20 adults with uncontrolled gout who have received a kidney transplant.

FDA Approved New Drug Application (NDA) for PROCYSBI Oral Granules: In February 2020, the FDA approved PROCYSBI Delayed-Release Oral Granules in Packets for adults and children one year of age and older living with nephropathic cystinosis. This new dosage form provides another administration option for patients, in addition to the currently available PROCYSBI capsules.

Two New Pipeline Programs Announced: In January 2020, the Company announced two new pipeline programs expected to begin in 2020: a TEPEZZA exploratory trial in diffuse cutaneous scleroderma and a proof of concept trial evaluating the impact of administering KRYSTEXXA over a shorter infusion duration.

Opened New Facility in South San Francisco: In November 2019, the Company opened a new office in South San Francisco. The 20,000 square-foot facility features laboratory space that will enable formulation and process development for manufacturing, as well as bioanalytical method development and other R&D functions. The Company expects to add new positions in bioanalysis, clinical research, pharmacology, manufacturing and business development in 2020.

Expanding the Company’s U.S. Operations: In February 2020, the Company closed the acquisition of its new U.S. headquarters in Deerfield, Ill. In line with the Company’s significant growth over the past three years, the new location will provide the Company the flexibility to accommodate its current U.S. operations as well as its anticipated future growth.

Research and Development Programs

TEPEZZA Diffuse Cutaneous Scleroderma Exploratory Trial: TEPEZZA is a fully human monoclonal antibody insulin-like growth factor-1 receptor (IGF-1R) inhibitor approved by the FDA for the treatment of TED. The Company is evaluating additional indications for TEPEZZA and expects to begin an exploratory trial in 2020 in diffuse cutaneous scleroderma, a rare fibrotic disease with no treatment options.

KRYSTEXXA MIRROR Randomized Clinical Trial: The Company is currently evaluating the coadministration of KRYSTEXXA with methotrexate to increase the complete response rate of KRYSTEXXA in the MIRROR placebo-controlled randomized clinical trial (RCT). The trial commenced in June 2019, and enrollment of 135 randomized patients is on track to complete mid-2020. The registrational trial is designed to enable the potential submission of results to the FDA to update the prescribing information. The MIRROR RCT follows an initial MIRROR open-label trial completed in 2019 that demonstrated a 79 percent complete response rate for patients using KRYSTEXXA with methotrexate. Methotrexate is the immunomodulator most used by rheumatologists and has been shown to reduce anti-drug antibody formation to biologic therapies when used in conjunction with these therapies.

KRYSTEXXA PROTECT Trial in Kidney Transplant Patients with Uncontrolled Gout: The Company is evaluating the effect of KRYSTEXXA on serum uric acid levels in kidney transplant patients with uncontrolled gout in its PROTECT clinical trial, initiated in October 2019. Kidney transplant patients have more than a tenfold increase in the prevalence of gout when compared to the general population, and literature suggests that persistently high serum uric acid levels can be associated with organ rejection. Managing uncontrolled gout is one of the most common and significant unmet needs of kidney transplant patients.

KRYSTEXXA Shorter-Infusion Duration Trial: The Company is initiating an open-label trial in mid-2020 to evaluate the impact of administering KRYSTEXXA over a significantly shorter infusion duration. Currently, KRYSTEXXA is infused over a two-hour or longer timeframe. A shorter infusion duration could meaningfully improve the experience and convenience for patients, physicians and sites of care.

Next-Generation Programs for Uncontrolled Gout: The Company is pursuing early-stage development programs for next-generation biologics for uncontrolled gout to support and sustain the Company’s market leadership in this area. These include HZN-003 and HZN-007, as well as a collaboration with HemoShear Therapeutics, LLC to discover new targets for gout.

Fourth-Quarter Financial Results

Note: For additional detail and reconciliation of non-GAAP financial measures to the most directly comparable GAAP financial measures, please refer to the tables at the end of this release.

Net Sales: Fourth-quarter 2019 net sales were $363.5 million, an increase of 2.3 percent.

Gross Profit: Under U.S. GAAP, the fourth-quarter 2019 gross profit ratio was 73.9 percent compared to 72.3 percent in the fourth quarter of 2018. The non-GAAP gross profit ratio in the fourth quarter of 2019 was 90.0 percent compared to 89.1 percent in the fourth quarter of 2018.

Operating Expenses: In the fourth quarter of 2019, research and development (R&D) expenses were 7.9 percent of net sales and selling, general and administrative (SG&A) expenses were 51.0 percent of net sales. Non-GAAP R&D expenses were 7.3 percent of net sales, and non-GAAP SG&A expenses were 44.3 percent of net sales.

Income Tax Rate: In the fourth quarter of 2019, the Company recorded a benefit of $555.9 million primarily related to an intra-company transfer of intellectual property assets, resulting in a tax rate on a GAAP basis of negative 1,507.0 percent. On a non-GAAP basis, fourth-quarter 2019 income tax expense was $11.7 million, resulting in a non-GAAP tax rate of 9.1 percent.

Net Income: On a GAAP basis in the fourth quarter of 2019, net income was $592.8 million. Fourth-quarter 2019 non-GAAP net income was $116.6 million.

Adjusted EBITDA: Fourth-quarter 2019 adjusted EBITDA was $139.9 million.

Earnings per Share: On a GAAP basis, diluted earnings per share (EPS) in the fourth quarter of 2019 and 2018 were $2.84 and $0.58, respectively. Non-GAAP diluted earnings per share in the fourth quarter of 2019 and 2018 were $0.56 and $0.67, respectively. The weighted average shares outstanding used to calculate fourth-quarter 2019 and 2018 non-GAAP diluted earnings per share were 211 million shares and 174 million shares, respectively. Given the recent share price appreciation, the Company’s $400 million dollars of exchangeable notes are approaching the point at which the Company would be able to redeem them for cash, ordinary shares or a combination of cash and ordinary shares. Based on current expectations, the Company is incorporating the potential conversion of the exchangeable notes into its fourth-quarter and full-year 2019 GAAP and non-GAAP diluted EPS calculations.

Fourth-Quarter Segment Results

Management uses net sales and segment operating income to evaluate the performance of the Company’s two segments, the orphan and rheumatology segment and the inflammation segment. While segment operating income contains certain adjustments to the directly comparable GAAP figures in the Company’s consolidated financial results, it is considered to be prepared in accordance with GAAP for purposes of presenting the Company’s segment operating results. Beginning with the first quarter of 2020, the Company is moving its medicine RAYOS, which is not an orphan medicine, to the inflammation segment, and the orphan and rheumatology segment is being renamed the orphan segment.

Beginning in 2019, the Company no longer recognizes revenue from RAVICTI and AMMONAPS sales outside of North America and Japan, nor from sales of LODOTRA. On Dec. 28, 2018, the Company divested the rights to RAVICTI and AMMONAPS outside of North America and Japan. AMMONAPS is known as BUPHENYL in the United States. In addition, effective Jan. 1, 2019, the RAYOS and LODOTRA license and supply agreements were amended, including the transfer of LODOTRA to Vectura Group plc. LODOTRA is known as RAYOS in the United States.

Fourth-quarter 2019 net sales of the orphan and rheumatology segment, the Company’s strategic growth segment, were $269.8 million, an increase of 14 percent over the prior year’s quarter, driven by growth of KRYSTEXXA and RAVICTI.

Fourth-quarter 2019 orphan and rheumatology segment operating income was $95.4 million, which includes the impact of investment in TEPEZZA pre-launch activities.

For the full-year 2019, KRYSTEXXA net sales of $342.4 million represented a 32 percent year-over-year increase, exceeding expectations.

In June 2019, the Company divested the rights to MIGERGOT.

Fourth-quarter 2019 net sales of the inflammation segment were $93.7 million and segment operating income was $44.0 million.

Cash Flow Statement and Balance Sheet Highlights

On a GAAP basis, operating cash flow was $191.4 million in the fourth quarter of 2019 and $426.3 million for the full year of 2019. Non-GAAP operating cash flow was $192.0 million in the fourth quarter of 2019 and $446.4 million for the full year of 2019.

The Company had cash and cash equivalents of $1.076 billion as of Dec. 31, 2019.

As of Dec. 31, 2019, the total principal amount of debt outstanding was $1.418 billion, consisting of $418 million in senior secured term loans due 2026, $600 million of senior notes due 2027 and $400 million of exchangeable senior notes due 2022. As of Dec. 31, 2019, net debt was $341.7 million and the net-debt-to-last-12-months adjusted EBITDA leverage (net leverage) ratio was 0.7 times, compared to 2.3 times at Dec. 31, 2018.

2020 Guidance

The Company expects full-year 2020 net sales to range between $1.40 billion and $1.42 billion, reflecting KRYSTEXXA full-year net sales growth of more than 25 percent and TEPEZZA full-year net sales of $30 million to $40 million. Full-year 2020 adjusted EBITDA is expected to range between $485 million and $500 million, reflecting significant investment in the U.S. launch of TEPEZZA and R&D pipeline programs to drive long-term growth.

Webcast

At 8 a.m. EST / 1 p.m. IST today, the Company will host a live webcast to review its financial and operating results and provide a general business update. The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.

Sierra Oncology to Hold Year End 2019 & 2020 Outlook Analyst & Investor Call

On February 26, 2020 Sierra Oncology, Inc. (SRRA), a late-stage drug development company focused on the registration and commercialization of momelotinib, a JAK1, JAK2 & ACVR1 inhibitor with a potentially differentiated therapeutic profile for the treatment of myelofibrosis, reported that its financial results for 2019 will be released on Tuesday, March 3 prior to market open (Press release, Sierra Oncology, FEB 26, 2020, View Source [SID1234554806]). The management team from Sierra, at 8:00 a.m. ET, will host a conference call to discuss the company’s progress made in 2019 and its plans for 2020.

Analyst and Investor Call Information

Date and Time: Tuesday, March 3 at 8:00 am ET

Domestic (Toll Free- US): 1-888-394-8218
International (Toll): 1-323-701-0225
Conference ID: 1052679
Webcast Link: www.sierraoncology.com
Direct Link: View Source

Call registration is available through the Sierra Oncology website at www.sierraoncology.com. An archive of the presentation will be accessible after the event through the Sierra Oncology website.

Exact Sciences to participate in March investor conferences

On February 26, 2020 Exact Sciences Corp. (Nasdaq: EXAS) reported that company management will participate in the following investor conferences and invited investors to participate by webcast (Press release, Exact Sciences, FEB 26, 2020, View Source [SID1234554805]).

Cowen 40th Annual Health Care Conference, Boston
Fireside Chat on Monday, March 2, 2020, at 1:30 p.m. EST

Barclays Global Healthcare Conference, Miami
Fireside Chat on Tuesday, March 10, 2020, at 3:50 p.m. EDT
The webcast can be accessed in the investor relations section of Exact Sciences’ website at www.exactsciences.com.

Portola Pharmaceuticals Reports Fourth Quarter and Full-Year 2019 Financial Results
and Provides Corporate Update

On February 26, 2020 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Portola Pharmaceuticals, FEB 26, 2020, View Source [SID1234554804]).

"2019 was a year of significant accomplishments for Portola with the launch of our Gen 2 formulation of Andexxa in the United States and the approval and launch of Ondexxya in Europe. In 2020 we have several catalysts that we expect to drive further adoption and growth worldwide," said Scott Garland, Portola’s president and chief executive officer. "This includes the presentation of new clinical data, enhanced education and support related to reimbursement, the initiation of our urgent surgery study, and continued execution of the Ondexxya launch in Europe. Combined with the robust growth in the Factor Xa inhibitor market and our other strategic initiatives, we are confident that Andexxa has significant long-term growth potential."
Quarter Ending December 31, 2019, and Full-Year 2019 Financial Results

Total global revenues for the fourth quarter of 2019 were $29.2 million compared with $15.3 million for the fourth quarter of 2018. This includes $28.4 million in net product revenues from sales of Andexxa/Ondexxya [coagulation factor Xa (recombinant), inactivated-zhzo], and $0.9 million in collaboration and license revenues. Total global revenues for the full year 2019 were $116.6 million compared with $40.1 million for the full year 2018. This includes $111.5 million in net product revenues from sales of Andexxa/Ondexxya, and $5.0 million in collaboration and license revenues.

Net loss attributable to Portola was $96.7 million, or $1.24 net loss per share for the fourth quarter of 2019, compared with a net loss of $88.5 million, or $1.34 net loss per share, for the same period in 2018. Net loss attributable to Portola was $290.7 million, or $4.06 net loss per share for the full year 2019, compared with a net loss of $350.2 million, or $5.31 net loss per share, for the full year 2018. This includes the effect of a $27.5 million charge taken in the fourth quarter of 2019 for Bevyxxa inventory and manufacturing related to the decision to discontinue the commercialization of Bevyxxa, and a $3.2 million impairment charge taken in the second quarter of 2019 related to the discontinuation of our SRX program.

Non-GAAP net loss for the fourth quarter of 2019 was $58.3 million, or a non-GAAP basic and diluted loss per share of $0.75. For the full year 2019, non-GAAP net loss was $198.2 million, or a non-GAAP basic and diluted loss per share of $2.77. Non-GAAP net loss and loss per share have been adjusted to exclude the Bevyxxa inventory and manufacturing charge and an impairment charge as well as stock-based compensation expenses. Please see the reconciliation of GAAP to non-GAAP financial measures at the end of this release for more details.

Total operating expenses for the fourth quarter of 2019 were $119.4 million compared with $102.5 million for the same period in 2018. Total operating expenses for the full year 2019 were $387.9 million, compared with $385.5 million for the full year 2018.

Non-GAAP total operating expenses, which excludes the Bevyxxa inventory and manufacturing charge and impairment charge as well as stock based compensation expenses, were $81.1 million for the fourth quarter of 2019, and $295.5 million for the full year 2019. Please see the reconciliation of GAAP to non-GAAP financial measures table at the end of this release for more details.

Research and development (R&D) expenses were $29.9 million for the fourth quarter of 2019 compared with $49.5 million for the fourth quarter of 2018. R&D expenses for the full year 2019 were $124.6 million, compared with $216.2 million for the full year 2018. The decrease in both periods is primarily due to the manufacturing costs for Andexxa Gen 2 being capitalized and no longer flowing through R&D.

Selling, general and administrative (SG&A) expenses for the fourth quarter 2019 were $60.0 million compared with $40.6 million for the fourth quarter of 2018. SG&A expenses for the full year 2019 were $218.9 million compared with $151.2 million for the full year 2018. The increase in both periods is due to commercial costs to support the launch of Andexxa, including the expansion of the field sales teams and support for the launch of Ondexxya in Europe.

Cost of sales (COS) for the fourth quarter of 2019 was $29.6 million compared to $12.4 million for the same period in 2018. COS for the full year 2019 was $44.4 million compared with $18.1 million for the full year 2018. The increase in both periods is due to a charge for Bevyxxa inventory and manufacturing as the Company winds down that program.

Please see the GAAP to non-GAAP reconciliation table at the end of this release for a detailed breakdown.
Cash, Cash Equivalents and Investments:

Cash, cash equivalents and investments at December 31, 2019, totaled $466.2 million, compared with $317.0 million as of December 31, 2018. During the fourth quarter, the Company drew down the remaining $62.5 million available under its $125.0 million loan agreement with HealthCare Royalty Partners (HCR) and investment funds manager Athyrium Capital Management, LP.
2020 Annual Financial Guidance
For the fiscal year 2020, Portola expects total R&D expenses to be between $105 million and $120 million, including stock-based compensation expenses of approximately $14 million. Portola expects total SG&A expenses to be between $235 million and $250 million, including stock-based compensation expenses of approximately $38 million.
Recent Achievements and Events

Completed an internal restructuring to align resources to drive Andexxa growth including the discontinuation of commercialization and partnering efforts for Bevyxxa. In addition, the Company has decided not to initiate the CELTIC-1 trial for the SYK/JAK inhibitor cerdulatinib until a partner is identified.

Initiated the single-arm urgent surgery study for Andexxa, ANNEXA-S.

Andexxa was highlighted as a first-line Factor Xa reversal option by the American College of Emergency Physicians in recommendations published in the Annals of Emergency Medicine, bringing the total number of guideline inclusions to 19.

Presented multiple abstracts related to cerdulatinib at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2019 Annual Meeting in December. The data demonstrated good tolerability and clinical response in patients with relapsed/refractory peripheral T-cell lymphoma (PTCL) and cutaneous T-cell lymphoma (CTCL), including a 52% overall response rate in patients with angioimmunoblastic T-cell lymphoma (AITL), and a 76% overall response rate in patients with follicular lymphoma treated with cerdulatinib in combination with rituximab.

Appointed 20-year industry veteran Rajiv Patni, M.D., as executive vice president and chief medical officer, with responsibility for leading Clinical Development, Clinical Operations, Medical Affairs, Regulatory Affairs, Biometrics, Pharmacovigilance and Project Management.

Launched a partnership with the American Heart Association supporting its hospital-focused quality improvement initiative for enhanced awareness and understanding of best care practices for hemorrhagic stroke.
Planned Upcoming Milestones

Present and publish clinical, research and HEOR studies supporting the adoption of Andexxa at medical meetings and in peer-reviewed journals throughout the year, starting with the presentation of three abstracts at the American College of Cardiology 2020 Annual Meeting in March.

Secure reimbursement coverage in the United Kingdom (early 2H 2020), Germany (2H 2020) and other Wave 1 European countries.

Continue launch of Ondexxya into Wave 2 countries in Europe, which include the additional EU5 countries of France, Spain, and Italy.

Conference Call Details
Portola will host a conference call today, Wednesday, February 26, 2020, at 4:30 p.m. ET, during which time management will discuss the fourth quarter and full-year 2019 financial results, updates on the U.S. and European launches of Andexxa/Ondexxya, and its operations. The live call can be accessed by phone by calling (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 6192918. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.

CYCLACEL PHARMACEUTICALS REPORTS FOURTH QUARTER AND FULL YEAR 2019 FINANCIAL RESULTS

On February 26, 2020 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported its financial results and business highlights for the fourth quarter and full year ended December 31, 2019 (Press release, Cyclacel, FEB 26, 2020, View Source [SID1234554803]). The Company’s net loss applicable to common shareholders for the three months and year ended December 31, 2019 was $2.4 million and $8.0 million, respectively. As of December 31, 2019, cash and cash equivalents totaled $11.9 million. Based on current spending, cash on hand provides the Company with sufficient resources to fund all planned operations including research and development through Q1 2021.

"Our fadraciclib (CYC065) CDK inhibitor is establishing a leadership position among MCL1 suppressing compounds in clinical development," said Spiro Rombotis, President and Chief Executive Officer. "After enrolling approximately 60 patients to date with multiple dosing schedules in three Phase 1 dose escalation studies in patients with relapsed or refractory (R/R) solid tumors and hematological malignancies, we have demonstrated safety, proof of mechanism by durable suppression of MCL1 and anticancer activity of fadraciclib as single agent and in combinations. Encouragingly, a heavily pretreated patient with MCL1 amplified endometrial cancer has received over 10 cycles of fadraciclib monotherapy achieved a confirmed partial response (PR) and a further reduction in her target tumor lesions of 73%. Based on data thus far, we are developing a precision medicine strategy to further evaluate fadraciclib as monotherapy and in combinations. In our Phase 1 trial of a combination of fadraciclib and venetoclax in patients with relapsed or refractory AML/MDS, we have enrolled 11 patients and reached dose level five and in CLL 3 patients and dose level two. Our clinical stage pipeline, comprising of fadraciclib, sapacitabine and CYC140, our PLK1 inhibitor, is a central element of our strategy of building an innovative pipeline addressing cancer resistance and DNA damage response."

Key Corporate Highlights

·CYC065-01 Phase 1 part 2 single agent i.v. – In November 2019, we reported anticancer activity in a heavily pretreated a patient with MCL1 amplified endometrial cancer who achieved a radiographically confirmed partial response (PR) after 4 cycles at 213mg. The patient remains on study after 10 cycles and shrinkage of her target tumor lesions has reached 73%. An additional patient with cyclin E amplified ovarian cancer achieved stable disease after 4 cycles at 213mg with 29% tumor shrinkage. We are expanding the 213mg dose level to recruit additional patients and determine the recommended Phase 2 dose.

·CYC065-01 Phase 1 part 3 single agent p.o. – We are evaluating an oral capsule form of fadraciclib in patients with advanced solid tumors and have enrolled two patients at 75mg and 150mg once daily. Pharmacokinetic (PK) data in the two patients demonstrated a predictable PK profile closely overlapping the i.v. form with encouraging exposure levels.

þ 200 Connell Drive, Suite 1500, Berkeley Heights, NJ 07922, USA Tel +1 908 517 7330 Fax +1 866 271 3466

¨ 1 James Lindsay Place, Dundee, DD1 5JJ, UK Tel +44 1382 206 062 Fax +44 1382 206 067

www.cyclacel.com – [email protected]

·CYC065-03 Phase 1 fadraciclib i.v. and venetoclax p.o. – We have dosed 11 heavily pretreated patients with R/R AML in five dose levels up to 200 mg/m2 in combination with the BCL2 inhibitor venetoclax. Evidence of anticancer activity has been observed in multiple patients with blast reductions in peripheral blood. Preclinical AML data demonstrated synergy of fadraciclib and venetoclax, suggesting that targeting both BCL2 and MCL1 may be more beneficial than inhibiting either protein alone.

·CYC065-02 Phase 1 fadraciclib i.v. and venetoclax p.o. – We have dosed 3 patients with R/R CLL in two dose levels up to 85 mg/m2 in combination with venetoclax. Evidence of anticancer activity has been observed in two patients who experienced reduction in lymph node size with one of them achieving MRD negative status. Preclinical CLL data demonstrated synergy of fadraciclib and venetoclax, suggesting that targeting both BCL2 and MCL1 may be more beneficial than inhibiting either protein alone.

·CYC682-11 Phase 1b/2 part 2 sapacitabine p.o. and venetoclax p.o.– We have enrolled 10 patients in two dose cohorts in our DNA Damage Response (DDR) program evaluating an oral combination of sapacitabine and venetoclax in patients with R/R AML/MDS. Sapacitabine is a nucleoside analogue that is active in AML and MDS R/R to prior therapy such as cytarabine or hypomethylating agents. Preclinical data demonstrated synergy of sapacitabine with BCL2 inhibition which may offer an effective, oral treatment regimen for patients who have failed front-line therapy.

·CYC140-01 Phase 1 CYC140 i.v. – We have enrolled 4 patients in our first-in-human, dose escalation study evaluating CYC140 in patients with advanced leukemias. CYC140 is a small molecule, selective polo-like-kinase 1 (PLK1) inhibitor that has demonstrated potent and selective target inhibition and high activity in xenograft models of human cancers.

More information on our clinical trials can be found at www.clinicaltrials.gov.

Key Business Objectives for 2020

·Report updated Phase 1 safety, PK and efficacy data for fadraciclib utilizing a frequent i.v. dosing schedule in patients with advanced solid cancers;

·Report initial safety and PK data from the Phase 1 study of an oral formulation of fadraciclib;

·Report initial safety and proof of concept data from the fadraciclib-venetoclax Phase 1 studies in R/R AML/MDS and CLL;

·Report initial data from the sapacitabine-venetoclax Phase 1b/2 study in patients with R/R AML/MDS;

·Report initial data from the CYC140 Phase 1 first-in-human study in R/R leukemias; and

·Report data from the Phase 1b/2 IST of sapacitabine-olaparib combination in patients with BRCA mutant metastatic breast cancer when reported by the investigators.

Financial Highlights

As of December 31, 2019, cash and cash equivalents totaled $11.9 million, compared to $17.5 million as of December 31, 2018. The decrease of $5.6 million was primarily due to net cash used in operating activities of $9.4 million, offset by $3.8 million of net cash provided by financing activities.

Revenues for the three months and year ended December 31, 2019 amounted to $0 compared to $0.2 million for the same periods in 2018. The 2018 revenue related to a collaboration, licensing and supply agreement with ManRos Therapeutics, entered into in June 2015.

Research and development expenses were $1.4 million and $4.7 million for the three months and year ended December 31, 2019 as compared to $1.1 million and $4.3 million for the same periods in 2018. Research and development expenses relating to the transcriptional regulation, CDK inhibitor program with fadraciclib increased by $0.5 million from $2.5 million for the year ended December 31, 2018 to $3.0 million for the year ended December 31, 2019, as the clinical evaluation of fadraciclib progressed. Research and development expenses relating to the DDR, sapacitabine program decreased by $0.4 million from $0.9 million for the year ended December 31, 2018 to $0.5 million for the year ended December 31, 2019, primarily as a result of expenditure related to clinical trial drug supply manufacturing in 2018.

General and administrative expenses for the three months and year ended December 31, 2019 were $1.4 million and $5.0 million respectively, compared to $1.5 million and $5.4 million for the same periods of the previous year.

Total other income, net for the three months and year ended December 31, 2019 were $0.1 million and $0.6 million, compared to $0.1 million and $0.9 million for the same periods of the previous year. The decrease of $0.3 million for the year ended December 31, 2019 is primarily related to a reduction in income received under an Asset Purchase Agreement with ThermoFisher Scientific.

United Kingdom research & development tax credits were $0.4 million and $1.3 million for the three months and year ended December 31, 2019 as compared to $0.4 million and $1.3 million for the same periods in 2018.

Net loss for the three months and year ended December 31, 2019 were $2.3 million and $7.8 million compared to $2.0 million and $7.3 million for the same periods in 2018.

The Company raised net proceeds of approximately $4.1 million during 2019, from its Common Stock Sales Agreement with H.C. Wainwright, which is now completed.

The Company estimates that cash resources of $11.9 million as of December 31, 2019 will fund currently planned programs through the first quarter of 2021.

Conference call information:

US/Canada call: (877) 493-9121 / international call: (973) 582-2750

US/Canada archive: (800) 585-8367 / international archive: (404) 537-3406

Code for live and archived conference call is 6761906.

For the live and archived webcast, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for 7 days.