On February 26, 2020 Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, provided business updates and reported financial results for the fourth quarter and full year of 2019 and highlighted pipeline progress (Press release, Moderna Therapeutics, FEB 26, 2020, View Source [SID1234554815]).

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New updates and recent progress include:

Infectious Diseases

Two of three dose cohorts in Phase 2 CMV vaccine (mRNA-1647) dose-confirmation study completed enrollment, and third and final cohort is more than 85% enrolled; first Phase 2 interim analysis expected in the third quarter of 2020; Moderna actively preparing for pivotal Phase 3 study, expected to begin in 2021
First clinical-grade batch of novel coronavirus vaccine (mRNA-1273) shipped and received by NIH for use in Phase 1 study in the U.S.; delivered from Company’s cGMP facility in 42 days from sequence selection
Rare Diseases

Dosing complete for 0.6 mg/kg dose cohort with steroid premedication in the Phase 1 study of antibody against chikungunya virus (mRNA-1944); first participant dosed in additional cohort with two doses of 0.3 mg/kg (without steroid premedication) given one week apart
First patient enrolled in Phase 1/2 MMA (mRNA-3704) study; actively recruiting patients at U.S. sites following a protocol amendment expanding the first cohort eligibility criteria to patients 8 years and older
Corporate Updates

On February 25, 2020, the Company received notice that the underwriters had exercised their option to purchase an additional $75 million in shares of common stock in connection with the recently completed public offering. The closing of this additional sale is expected to occur on or about February 26, 2020, subject to the satisfaction of customary closing conditions.
"The Moderna team continues to execute our strategy, including our CMV Phase 2 study enrolling ahead of plan, shipping the coronavirus Phase 1 clinical materials to NIH/NIAID in just 42 days, and announcing five new development candidates in the last two months," said Stéphane Bancel, Moderna’s Chief Executive Officer. "We have up to $2 billion of capital to invest in the Company, a great team, a powerful mRNA platform and a state-of-the-art manufacturing site. I am more energized than ever about our future."

Moderna currently has 24 mRNA development candidates in its portfolio with 12 in clinical studies. Across Moderna’s pipeline, more than 1,700 participants have been enrolled in clinical studies. The Company’s updated pipeline can be found at www.modernatx.com/pipeline. Moderna and collaborators have published more than 40 peer-reviewed papers, including 21 in 2019.

Summary of Program Highlights by Modality

Core Modalities

Prophylactic Vaccines: Moderna is developing vaccines against viral diseases where there is unmet medical need – including complex vaccines with multiple antigens for common diseases, as well as vaccines against threats to global public health. The Company’s global public health portfolio is focused on epidemic and pandemic diseases for which funding has been sought from government and non-profit organizations.

Infections transmitted from mother to baby

Cytomegalovirus (CMV) vaccine (mRNA-1647): The first (50 µg) and second (100 µg) cohorts of the Phase 2 dose-confirmation study of mRNA-1647 have completed enrollment, and the third and final cohort (150 µg) is more than 85% enrolled. In February, Moderna announced that the first interim analysis of this Phase 2 study is expected in the third quarter of 2020. In January, Moderna announced positive seven-month interim data after the third and final vaccination from the Phase 1 study of mRNA-1647. Manufacturing and planning are underway for the pivotal Phase 3 study, which is designed to evaluate the efficacy of mRNA-1647 against primary CMV infection in a population that includes women of childbearing age and is expected to start in 2021. Moderna owns worldwide commercial rights for mRNA-1647.
Zika virus (mRNA-1893): The 10 µg, 30 µg and 100 µg cohorts in the Phase 1 study of mRNA-1893 have completed enrollment. This development candidate is being developed in collaboration with the U.S. Biomedical Advanced Research and Development Authority (BARDA) within the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services. Moderna owns worldwide commercial rights to mRNA-1893.
Vaccines against respiratory infections

Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3) vaccine (mRNA-1653): The Phase 1b age de-escalation study of mRNA-1653 is ongoing. Moderna previously announced positive data from the second pre-planned interim analysis of the Phase 1 study of mRNA-1653. Moderna owns worldwide commercial rights to mRNA-1653.
Pediatric respiratory syncytial virus (RSV) vaccine (mRNA-1345): mRNA-1345 is a vaccine against respiratory syncytial virus (RSV) in young children encoding for a prefusion F glycoprotein, which elicits a superior neutralizing antibody response compared to the postfusion state. The Company intends to combine mRNA-1345 with mRNA-1653, its vaccine against hMPV and PIV3, to create a combination vaccine against RSV, hMPV and PIV3. There is no approved vaccine for RSV. Moderna owns worldwide commercial rights to the combined mRNA-1345/mRNA-1653 vaccine.
Respiratory syncytial virus (RSV) vaccine (mRNA-1172 or V172): The Phase 1 study of mRNA-1172 led by Merck is ongoing. Moderna has licensed worldwide commercial rights to mRNA-1172 to Merck.
Novel coronavirus (SARS CoV-2) vaccine (mRNA-1273): The first clinical-grade batch of mRNA-1273 has been shipped from Moderna facility to the NIH for use in a Phase 1 study in the U.S. mRNA-1273 was delivered from the Company’s cGMP facility in 42 days from sequence selection. NIH plans to conduct the Phase 1 clinical trial in the U.S. There is no approved vaccine for novel coronavirus.
Influenza H7N9 vaccine (mRNA-1851): Discussions regarding funding the Company’s influenza H7N9 vaccine program through approval are ongoing.
Vaccines against highly prevalent viral infections

Epstein-Barr virus (EBV) vaccine (mRNA-1189): mRNA-1189 is a vaccine against Epstein-Barr virus (EBV) containing five mRNAs that encode viral proteins (gp350, gB, gp42, gH and gL) in EBV. Similar to Moderna’s CMV vaccine (mRNA-1647), the viral proteins in mRNA-1189 are expressed in their native membrane-bound form for recognition by the immune system. There is no approved vaccine for EBV. Moderna owns worldwide commercial rights to mRNA-1189.
Systemic Secreted & Cell Surface Therapeutics: In this modality, mRNA is delivered systemically to create proteins that are either secreted or expressed on the cell surface.

Antibody against the chikungunya virus (mRNA-1944): Dosing is complete for the 0.6 mg/kg cohort with steroid premedication in the Phase 1 study evaluating escalating doses of mRNA-1944 administered via intravenous infusion in healthy adults. The first participant has been dosed in the additional cohort with two doses of 0.3 mg/kg (without steroid premedication) given one week apart. In September 2019, Moderna announced positive interim data from the first analysis of safety and activity in the Phase 1 study. Moderna owns worldwide commercial rights to mRNA-1944.
IL-2 (mRNA-6231): mRNA-6231 is an mRNA encoding for a long-acting tolerizing IL-2. This new autoimmune development candidate is designed to preferentially activate and expand the regulatory T cell population. The Company plans to conduct a Phase 1 study of mRNA-6231 in healthy adult volunteers. mRNA-6231 uses the same LNP formulation as mRNA-1944. The Phase 1 study of mRNA-6231 will be the first clinical demonstration of subcutaneous administration of this delivery technology. Moderna owns worldwide commercial rights to mRNA-6231.
PD-L1 (mRNA-6981): mRNA-6981 is an mRNA encoding for PD-L1. This new autoimmune development candidate is designed to augment cell surface expression of PD-L1 on myeloid cells to provide co-inhibitory signals to self-reactive lymphocytes. As an initial step to addressing a range of autoimmune indications, the Company intends to pursue proof-of-concept in a Phase 1 study of mRNA-6981 in type 1 autoimmune hepatitis (AIH), a condition that involves liver inflammation and can lead to cirrhosis and liver failure. mRNA-6981 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-6981.
Relaxin (AZD7970): Partnered with AstraZeneca, AZD7970 is in preclinical development for the treatment of heart failure. Under the terms of the collaboration, AstraZeneca would sponsor the Phase 1 trial to assess safety, tolerability and duration of systemic exposure to the Relaxin protein. Moderna shares worldwide commercial rights to AZD7970 with AstraZeneca.
Fabry disease (mRNA-3630): Individuals with Fabry disease have a deficiency in the α-GAL enzyme resulting in a reduced or complete inability to metabolize glycosphingolipids in lysosomes. mRNA-3630 aims to instruct cells to produce α-GLA both locally in multiple affected tissues, and to secrete it into circulation from organs such as the liver for delivery to distal tissues. mRNA-3630 is in preclinical development. Moderna owns worldwide commercial rights to mRNA-3630.
Exploratory Modalities

Cancer Vaccines: These programs focus on stimulating a patient’s immune system with antigens derived from tumor-specific mutations to enable the immune system to elicit a more effective anti-tumor response.

Personalized cancer vaccine (PCV) (mRNA-4157): The randomized Phase 2 study investigating a 1 mg dose of mRNA-4157 in combination with Merck’s pembrolizumab (KEYTRUDA), compared to pembrolizumab alone, for the adjuvant treatment of high-risk resected melanoma is ongoing. The Phase 1 study is ongoing. Moderna shares worldwide commercial rights to mRNA-4157 with Merck.
KRAS vaccine (mRNA-5671 or V941): The Phase 1 open-label, multi-center study to evaluate the safety and tolerability of mRNA-5671 both as a monotherapy and in combination with pembrolizumab, led by Merck, is ongoing. Moderna shares worldwide commercial rights to mRNA-5671 with Merck.
Intratumoral Immuno-Oncology: These programs aim to drive anti-cancer T cell responses by injecting mRNA therapies directly into tumors.

OX40L (mRNA-2416): The first patient has been dosed in the Phase 1 dose escalation cohort of mRNA-2416 in combination with durvalumab (IMFINZI). The Company has removed the top dose of 8 mg in this cohort based on limitations due to the size of ovarian lesions. Moderna owns worldwide commercial rights to mRNA-2416.
OX40L/IL-23/IL-36γ (Triplet) (mRNA-2752): The Phase 1 trial evaluating mRNA-2752 as a single agent and in combination with durvalumab in patients with advanced solid tumor malignancies and lymphoma is ongoing. mRNA-2752 is an investigational mRNA immuno-oncology therapy that encodes a novel combination of three immunomodulators. Moderna owns worldwide commercial rights to mRNA-2752.
IL-12 (MEDI1191): The Phase 1 open-label, multi-center study of intratumoral injections of MEDI1191 alone and in combination with durvalumab in patients with advanced solid tumors, led by AstraZeneca, is ongoing. MEDI1191 is an mRNA encoding for IL-12, a potent immunomodulatory cytokine. Moderna shares worldwide commercial rights to MEDI1191 with AstraZeneca.
Localized Regenerative Therapeutics: Localized production of proteins has the potential to be used as a regenerative medicine for damaged tissues.

VEGF-A (AZD8601): The Phase 2a study of AZD8601 for VEGF-A for ischemic heart disease in patients undergoing coronary artery bypass grafting (CABG) surgery with moderately impaired systolic function, led by AstraZeneca, is ongoing. Moderna has licensed worldwide commercial rights to AZD8601 to AstraZeneca.
Systemic Intracellular Therapeutics: These programs aim to deliver mRNA into cells within target organs as a therapeutic approach for diseases caused by a missing or defective protein.

Methylmalonic acidemia (MMA) (mRNA-3704): The first patient has been enrolled in the Phase 1/2 open-label, dose escalation study evaluating the safety and tolerability of escalating doses of mRNA-3704 administered via intravenous infusion in patients with isolated methylmalonic acidemia (MMA) due to MUT deficiency. The patient has entered an observational period prior to treatment, which evaluates the patient’s baseline disease prior to starting the treatment period. The Company is planning to initiate several sites outside the U.S. and has thus far received Medicines and Healthcare products Regulatory Agency (MHRA) approval in the U.K. The objectives of this study are to evaluate safety and tolerability, assess the pharmacodynamic response and characterize the pharmacokinetic profile of mRNA-3704. This is Moderna’s first rare disease program to begin clinical trial enrollment. mRNA-3704 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-3704.
Propionic acidemia (PA) (mRNA-3927): Study start-up in the U.S. is ongoing for the open-label, multi-center Phase 1/2 study of multiple ascending doses of mRNA-3927 in primarily pediatric patients with PA. The objectives of this study are to evaluate the safety and tolerability of mRNA-3927 administered via IV infusion, assess the pharmacodynamic response as assessed by changes in plasma biomarkers and characterize the pharmacokinetic profile of mRNA-3927. mRNA-3927 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-3927.
MMA and PA Natural History Study (MaP): This is a global, multi-center, non-interventional study for patients with confirmed diagnosis of MMA due to MUT deficiency or PA and is designed to identify and correlate clinical and biomarker endpoints for these disorders. Enrollment in the study has been completed.
Phenylketonuria (PKU) (mRNA-3283): Individuals with PKU have a deficiency in phenylalanine hydroxylase (PAH) resulting in a reduced or complete inability to metabolize the essential amino acid phenylalanine into tyrosine. mRNA-3283 encodes human PAH to restore the deficient or defective intracellular enzyme activity in patients with PKU. mRNA-3283 is in preclinical development. Moderna owns worldwide commercial rights to mRNA-3283.
Glycogen storage disease type 1a (GSD1a) (mRNA-3745): Individuals with GSD1a have a deficiency in glucose-6-phosphatase resulting in pathological blood glucose imbalance. mRNA-3745 is an IV-administered mRNA encoding human G6Pase enzyme, designed to restore deficient or defective intracellular enzyme activity in patients with GSD1a. mRNA-3745 is in preclinical development. Moderna owns worldwide commercial rights to mRNA-3745.
Information about each development candidate in Moderna’s pipeline, including those discussed in this press release, can be found on the investor relations page of its website: investors.modernatx.com.

Corporate Updates

Public offering, strategic alliances and available grant funding: After the closing of the underwriters’ option to purchase additional shares, the Company will have raised $550 million in net proceeds from the recent financing announced in early February 2020.
The Company has established a wide range of strategic alliances with leading biopharmaceutical companies, as well as grants from government-sponsored and private organizations focused on global health initiatives. As of December 31, 2019, Moderna had up to $185 million in additional funding available from grants (including amounts not yet committed)1.

With the offering proceeds, access to additional grant funding, and cash and investments of $1.26 billion as of December 31, 2019, Moderna has access to up to $2 billion in capital to invest in the business.

Shareholder letter: Moderna CEO Stéphane Bancel published a letter to shareholders on January 6, 2020.
Key 2020 Investor and Analyst Event Dates

Manufacturing & Digital Day – March 4 at Moderna’s Norwood, MA facility
Vaccines Day – April 14 in New York City
Science Day – June 2 in New York City
R&D Day – September 17 in New York City
Fourth Quarter and Full Year 2019 Financial Results (Unaudited)

Cash Position: Cash, cash equivalents and investments as of December 31, 2019 and 2018 were $1.26 billion and $1.69 billion, respectively.
Net Cash Used in Operating Activities: Net cash used in operating activities was $459.0 million for the year ended December 31, 2019 compared to $330.9 million for the year ended December 31, 2018.
Cash Used for Purchases of Property and Equipment: Cash used for purchases of property and equipment decreased $74.2 million, or 70.2%, to $31.6 million for the year ended December 31, 2019 from $105.8 million for the year ended December 31, 2018. Of these amounts, cash disbursements specifically related to Moderna Technology Center (MTC) manufacturing facility in Norwood, MA were $14.6 million and $94.5 million for the years ended December 31, 2019 and 2018, respectively. Our MTC manufacturing facility opened in July 2018.
Revenue: Total revenue was $14.1 million for the fourth quarter of 2019 compared to $35.4 million for the fourth quarter of 2018. Total revenue was $60.2 million for the year ended December 31, 2019 compared to $135.1 million for the year ended December 31, 2018. The decreases in both periods were mainly due to lower collaboration revenue across all our strategic alliances, particularly AstraZeneca and Merck, driven by our adoption of ASC 606 and the completion of the initial four-year research period under the 2016 Merck Agreement.
Research and Development Expenses: Research and development expenses were $118.8 million for the fourth quarter of 2019 compared to $150.4 million for the fourth quarter of 2018. Research and development expenses were $496.3 million for the year ended December 31, 2019 compared to $454.1 million for the year ended December 31, 2018. The decrease in the fourth quarter was primarily due to a decrease in our in-licensing payments to Cellscript, LLC and its affiliate, and a reduction of our lab supplies and materials costs. The increase for the year ended December 31, 2019 was primarily due to an increase in personnel related cost, including stock-based compensation, and an increase in clinical trial and manufacturing costs, mainly driven by an increase in the number of employees and costs supporting research and development programs.
General and Administrative Expenses: General and administrative expenses were $25.9 million for the fourth quarter of 2019 compared to $38.0 million for the fourth quarter of 2018. General and administrative expenses were $109.6 million for the year ended December 31, 2019 compared to $94.3 million for the year ended December 31, 2018. The decrease in the fourth quarter was primarily due to a decrease in stock-based compensation, mainly attributable to certain performance-based equity awards with vesting or commencement contingent on our initial public offering in 2018. The increase for the year ended December 31, 2019 was primarily due to an increase in insurance, consulting and outside services, and facility related costs, primarily driven by an increase in the number of employees and costs in support of being a public company.
Net Loss: Net loss was $124.2 million for the fourth quarter of 2019 compared to $141.4 million for the fourth quarter of 2018. Net loss was $514.0 million for the year ended December 31, 2019 compared to $384.7 million for the year ended December 31, 2018.
Reiterating Financial Guidance

Moderna expects net cash used in operating activities and for purchases of property and equipment in 2020 to be similar to 2019, between $490 million and $510 million.
Investor Call and Webcast Information

Moderna will host a live conference call and webcast at 8:00 a.m. ET on Wednesday, February 26, 2020. To access the live conference call, please dial 866-922-5184 (domestic) or 409-937-8950 (international) and refer to conference ID 3639288. A webcast of the call will also be available under "Events and Presentations" in the Investors section of the Moderna website at investors.modernatx.com. The archived webcast will be available on Moderna’s website approximately two hours after the conference call and will be available for 30 days following the call.

On February 26, 2020 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, announced today that the Company has scheduled the release of its fourth quarter and full year 2019 financial results before market opens on Friday, February 28, 2020 (Press release, Sangamo Therapeutics, FEB 26, 2020, View Source [SID1234554814]). The press release will be followed by a conference call at 8:00 a.m. ET, which will be open to the public via telephone and webcast. During the conference call, the Company will review its financial results and provide a business update.

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The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 4609858. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 4609858.

On February 26, 2020 Momenta Pharmaceuticals, Inc. (Nasdaq: MNTA), a biotechnology company focused on discovering and developing novel biologic therapeutics to treat rare immune-mediated diseases, reported its financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Momenta Pharmaceuticals, FEB 26, 2020, View Source [SID1234554808]).

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"2019 was a transformative year for Momenta as the power of our pipeline began to show itself," said Craig A. Wheeler, President and Chief Executive Officer of Momenta Pharmaceuticals. "Thanks to our talented research team, we are also expanding our pipeline, starting with the nomination of M267, our new SIFbody development candidate earlier this year."

Fourth Quarter 2019 Highlights, Recent Events and Anticipated Upcoming Milestones

Novel Therapeutics Pipeline:

M254 (hsIgG): a hypersialylated immunoglobulin designed as a high potency alternative for intravenous immunoglobulin (IVIg)

•The Company’s multi-part Phase 1/2 clinical trial in idiopathic thrombocytopenic purpura (ITP) is progressing through Part B, which is evaluating M254 compared to IVIg in a single ascending dose (SAD) cohort of ITP patients. In January 2020, Momenta announced interim results, featuring early favorable responses to M254 (5/6 patients on treatment exhibited platelet counts ≥ 50 x 109/L). Based on these data, the Company is expanding Part B to include at least one additional lower dose cohort and to augment the number of patients in existing cohorts. Enrollment for the study is ongoing, with additional data from Part B to be submitted for presentation at a medical conference in the second quarter of 2020.

•The Company plans to launch a Phase 2 study of M254 in chronic inflammatory demyelinating polyneuropathy (CIDP) in the fourth quarter of 2020.

Nipocalimab (M281): a fully human anti-neonatal Fc receptor (FcRn) aglycosylated immunoglobulin G (IgG1) monoclonal antibody (mAb)

•The Company has completed its target enrollment in Vivacity-MG, the Phase 2 clinical study of nipocalimab in generalized myasthenia gravis (gMG). The Company expects to report top-line data in the third quarter of 2020.
•Unity, the Company’s global multi-center Phase 2 clinical study of nipocalimab in hemolytic disease of the fetus and newborn (HDFN), is enrolling well. The Company expects to report top-line data from this study in 2021.
•Energy Study, the Company’s adaptive Phase 2/3 clinical study of nipocalimab in warm autoimmune hemolytic anemia (wAIHA) is enrolling patients and the Company is activating clinical sites in both the United States and European Union. Top-line data are expected from this study around the end of 2021.

M230 (CSL730): a recombinant Fc multimer being developed in collaboration with CSL

•A Phase 1 clinical program to evaluate the safety and tolerability of M230 in healthy volunteers is continuing. Momenta’s partner, CSL, plans to introduce a subcutaneous formulation into the Phase 1 program this year.

Momenta’s SIFbody platform combines multiple Fc’s with antibody fabs to optimally activate Fc and complement effector function and effectively deplete target cells.

•In January 2020, Momenta nominated M267, a SIFbody candidate targeting CD38, for clinical development. Pre-clinical data suggest this candidate has the potential to be a best-in-class therapeutic to target CD38 expressing cells, which are prevalent in plasmacyte-mediated diseases such as multiple myeloma, AL amyloidosis and rare, autoantibody-mediated diseases.
•The Company plans to initiate IND-enabling studies for this candidate in 2020.

Legacy Products:

GLATOPA 20 mg and 40 mg: FDA approved generic versions of COPAXONE 20 mg and 40 mg, developed and commercialized in collaboration with Sandoz

•In the fourth quarter of 2019, Momenta recorded $7.9 million in product revenue from Sandoz’s sales of GLATOPA products.

M710: a proposed biosimilar to EYLEA (aflibercept) candidate being developed in collaboration with Mylan

•Mylan continues its pivotal clinical trial in patients with diabetic macular edema to compare safety, efficacy and immunogenicity of M710 with EYLEA. Mylan expects to target U.S. submission in 2021.

Corporate:

•In January 2020, Momenta announced the appointment of Young Kwon, Ph.D. as Chief Financial and Business Officer. Dr. Young previously served as Chief Business Officer at the Company.

•In December 2019, the Company announced the appointment of Jane F. Barlow, M.D., M.P.H., M.B.A. to its Board of Directors.

•In December 2019, Momenta announced the closing of a public offering of 16.7 million shares of its common stock at the price of $15.50 per share. Net proceeds from the offering were $244.2 million.

•In December 2019, Momenta and Sandoz entered into a settlement agreement with The Hospital Authority of Metropolitan Government of Nashville and Davidson County, Tennessee, d/b/a Nashville General Hospital, or NGH, resolving all pending litigation between the parties related to Enoxaparin Sodium Injection, an FDA-approved, substitutable generic LOVENOX, which Momenta developed in collaboration with Sandoz. As a result of the settlement, the Company agreed to pay an aggregate of $35.0 million as consideration for the release of all alleged claims.

Fourth Quarter and Full Year 2019 Financial Results

Revenue:

In the fourth quarter of 2019, the Company recorded $7.9 million in product revenue from Sandoz’s sales of GLATOPA, compared to $10.8 million for the same period in 2018. For the year ended December 31, 2019, the Company recorded $19.1 million in product revenue from Sandoz’s sales of GLATOPA, compared to $39.7 million for the same period in 2018. The decrease in product revenue of $2.9 million, or 27%, from the fourth quarter of 2018 to the fourth quarter of 2019 was primarily due to lower net sales of GLATOPA driven by competition. The decrease in product revenue of $20.6 million, or 52%, from the year ended 2018 to the year ended 2019 was primarily due to lower net sales of GLATOPA driven by competition, a $1.5 million legal settlement payment to Teva Pharmaceuticals Industries Ltd. and related entities in the first quarter of 2019, representing Momenta’s 50% share, and $1.7 million received by Momenta in the third quarter of 2018 for the Pfizer settlement.

Research and development revenue for the fourth quarter of 2019 was $0.3 million compared to $32.1 million for the same quarter in 2018. For the year ended December 31, 2019, research and development revenue was $4.8 million compared to $35.9 million for the same period in 2018. The decrease in research and development revenue of $31.8 million, or 99.1%, and $31.1 million, or 87%, from the fourth quarter of 2018 to the fourth quarter of 2019, and from the year ended 2018 to the year ended 2019, respectively, was primarily due to $28.4 million of revenue recognized related to Mylan’s upfront payment of $45.0 million during the fourth quarter of 2018 and lower reimbursement revenue for GLATOPA expenses in 2019.

Total revenue for the fourth quarter of 2019 was $8.2 million, compared to $42.8 million for the same period in 2018. For the year ended December 31, 2019, total revenue was $23.9 million, compared to $75.6 million for the same period in 2018.

Operating Expenses:

Research and development expenses for the fourth quarter of 2019 were $38.3 million, compared to $28.7 million for the same period in 2018. The increase of $9.6 million, or 33%, was primarily due to an increase in manufacturing and clinical trial costs for nipocalimab and M254, offset in part by lower personnel costs following the Company’s workforce reduction in the fourth quarter of 2018 and a reduction in lease costs. For the year ended December 31, 2019, research and development expenses were $144.5 million, compared to $124.0 million for the same period in 2018. The increase of $20.5 million, or 17%, was primarily due to an increase in manufacturing and clinical trial costs for nipocalimab and M254, offset partially by a decrease in our share of CSL collaboration costs, lower personnel costs following the Company’s workforce reduction in the fourth quarter of 2018 and a reduction in lease costs.

General and administrative expenses for the fourth quarter of 2019 were $58.9 million, compared with $21.5 million for the same period in 2018. The increase of $37.4 million, or 174%, was primarily due to $35.0 million related to a settlement agreement with Nashville General Hospital related to Enoxaparin Sodium Injection. For the year ended December 31, 2019, general and administrative expenses were $149.8 million, compared to $85.1 million for the same period in 2018. The increase of $64.7 million, or 76%, was primarily due to $35.0 million related to the settlement agreement with Nashville General Hospital, $21.0 million paid to Amphastar Pharmaceuticals in June 2019, reflecting the Company’s portion of the required settlement payments related to Enoxaparin Sodium Injection, increased depreciation of $4.8 million associated with a change in the estimated useful life of certain leasehold improvements in the fourth quarter of 2018, increased share-based compensation expense of $4.1 million, driven primarily by expense recognized on performance-based restricted stock units in the fourth quarter of 2019, and increased consultant spend of $4.2 million. These increases were partially offset by decreased personnel costs, including salaries and share-related benefits, of $3.9 million due to the workforce reduction announced in October 2018.

In July 2019, the Company entered into an amendment to its office and laboratory space lease at 320 Bent Street in Cambridge, Massachusetts, reducing the Company’s footprint at this location. During the year ended December 31, 2019, the Company recognized a non-cash gain of $13.7 million, reflecting the reduction in the lease liability and the related right-of-use asset.

Total GAAP operating expenses for the fourth quarter of 2019 were $95.5 million, compared to $52.5 million for the same period in 2018. For the year ended December 31, 2019, total GAAP operating expenses were $322.0 million, compared to $256.9 million for the same period in 2018.

Fourth quarter non-GAAP operating expense was $86.9 million. Full year 2019 non-GAAP operating expense was $298.5 million. Non-GAAP operating expense is total operating expenses, less restructuring costs, stock-based compensation expense and collaborative reimbursement revenues. See "Non-GAAP Financial Information and Other Disclosures" and the table below entitled "Reconciliation of GAAP Results to Non-GAAP Financial Measures" for a reconciliation of GAAP operating expense to non-GAAP operating expense.

Net Income (Loss):

The Company reported a net loss of $86.7 million, or $0.85 per share for the fourth quarter of 2019, compared to a net loss of $8.2 million, or $0.10 per share for the same period in 2018. For the year ended December 31, 2019, the Company reported a net loss of $290.1 million, or $2.92 per share compared to a net loss of $176.1 million, or $2.26 per share for 2018.

Cash Position:

At December 31, 2019, the Company had $545.1 million in cash, cash equivalents and marketable securities, reflecting the December 2019 common stock financing compared to $325.9 million at September 30, 2019.

2020 Financial Guidance

Momenta provides non-GAAP operating expense guidance, which it believes can enhance an overall understanding of its financial performance when considered together with GAAP financial measures. Refer to the section of this press release below entitled "Non-GAAP Financial Information and Other Disclosures" for further discussion of this subject.

Non-GAAP operating expense is total operating expenses, less stock-based compensation expense, restructuring expense and collaborative reimbursement revenues. Momenta is providing full-year non-GAAP operating expense guidance of $220 – $240 million for 2020.

Non-GAAP Financial Information and Other Disclosures

Momenta uses a non-GAAP financial measure, non-GAAP operating expense, to provide operating expense guidance. Momenta believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Momenta’s operating performance as it excludes non-cash stock compensation expense, restructuring expense and collaborative reimbursement revenues. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP total operating expense and should not be considered a measure of Momenta’s liquidity. Instead, non-GAAP operating expense should only be used to supplement an understanding of Momenta’s operating results as reported under GAAP. Momenta has not provided GAAP reconciliation for its forward-looking non-GAAP annual operating expense because Momenta cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the forward-looking non-GAAP financial measure. The Company does not expect restructuring expense and collaboration reimbursement revenue to be material.

Conference Call Information

Management will host a conference call and webcast today at 8:30 am ET to discuss these results and provide an update on the Company. A live webcast of the conference call may be accessed on the "Investors" section of the Company’s website, www.momentapharma.com. Please go to the site at least 15 minutes prior to the call in order to register, download, and install any necessary software. An archived version of the webcast will be posted on the Momenta website approximately two hours after the call.

To access the call you may also dial (866) 209-9686 (domestic) or (825) 312-2288 (international) prior to the scheduled conference call time and provide the access code 8677153.

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.

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.

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!

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.