On February 25, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported financial results for the fourth quarter and full year ending December 31, 2020 and provided a corporate update (Press release, PTC Therapeutics, FEB 25, 2021, View Source [SID1234575666]).

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"Despite the challenges of the pandemic, PTC has been able to make significant progress in moving our pipeline forward and has been able to continue to bring therapies to our patients throughout 2020," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I am very proud of our team and their abilities to execute on our 2020 goals even in such turbulent times. I am confident that we will continue to progress on our 2021 goals that will create value for all of our stakeholders."

Key 2020 Corporate Highlights:

Strong continued revenue growth for the DMD franchise, with total net product revenue of $331 million for Translarna (ataluren) and Emflaza (deflazacort) in 2020.
Translarna growth was driven by broader uptake due to new patients in existing geographies, geographic expansion and label updates.
Emflaza experienced strong 38% year-over-year revenue growth in 2020 due to increased new prescriptions and high compliance.
Evrysdi (risdiplam) was approved by the FDA in August 2020 for treatment of spinal muscular atrophy (SMA) patients aged 2 months and older. A strong global launch is under way, leading to increased patient uptake across all disease subtypes. Evrysdi is a product of a collaboration between PTC, Roche, and the SMA Foundation.
PTC initiated five clinical trials in 2020, three of which are registration-directed clinical studies:
The MIT-E Phase 2/3 trial with vatiquinone for mitochondrial epilepsy with data anticipated in the third quarter of 2022.
The MOVE-FA Phase 3 trial with vatiquinone for Friedreich ataxia with data anticipated in 2023.
The FITE19 Phase 2/3 clinical trial for PTC299 in patients with COVID-19.
A healthy volunteer study for PTC857, the second Bio-e compound.
A healthy volunteer study for PTC518 for the Huntington disease program from the splicing platform.
Submitted a Marketing Authorization Application (MAA) to European Medicines Agency (EMA) for PTC-AADC, a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency.
PTC strengthened its balance sheet with a $650 million upfront payment for a portion of the Evrysdi royalties.
2021 Potential Value-Creating Milestones:

Results from Phase 1 healthy volunteer study of PTC518 for Huntington disease program from the splicing platform are expected in the first half.
Data from the healthy volunteer study for PTC857 are expected in the first half.
PTC expects an opinion on the PTC-AADC MAA from the EMA’s Committee for Medicinal Products for Human Use (CHMP) in the second quarter.
PTC is on track for a submission of a Biologics License Application (BLA) to the FDA in the second quarter for PTC-AADC.
PTC anticipates an opinion from the CHMP for Evrysdi in the first quarter.
A registration-directed study, APHENITY, for PTC923 in patients with phenylketonuria (PKU) is expected to initiate mid-year.
The second stage of the FITE19 Phase 2/3 clinical trial to assess PTC299 in patients with COVID-19 has been initiated. Data from this study is expected in the second half of the year.
PTC expects to dose the first patient with PTC-FA gene therapy for Friedreich ataxia before year end.
Results from the ongoing clinical trials evaluating PTC596 in Leiomyosarcoma (LMS) and Diffuse Intrinsic Pontine Glioma (DIPG) are expected by year end.
Fourth Quarter and Full Year 2020 Financial Highlights:

Total revenues were $118.9 million for the fourth quarter of 2020, compared to $96.5 million for the fourth quarter of 2019. Total revenues were $380.8 million for the full year 2020, compared to $307.0 million for the full year 2019.
Total revenue includes net product revenue across the commercial portfolio of $107.3 million for the fourth quarter of 2020 and $333.4 million for full year 2020, and collaboration and royalty revenue of $11.6 million for the fourth quarter of 2020 and $47.4 million for full year 2020.
Translarna net product revenues were $69.4 million for the fourth quarter of 2020, compared to $48.4 million for the fourth quarter of 2019. Translarna net product revenues were $191.9 million for the full year 2020, compared to $190.0 million for the full year 2019. In October 2020, PTC secured a purchase agreement for Translarna with the Ministry of Health in Brazil specifying two shipments, both of which were fulfilled in 2020.
Emflaza net product revenues were $36.8 million for the fourth quarter of 2020, compared to $32.7 million for the fourth quarter of 2019. Emflaza net product revenues were $139.0 million for the full year 2020, compared to $101.0 million for the full year 2019.
Roche reported Evrysdi 2020 year-to-date sales of approximately CHF 55 million. During the third quarter of 2020, the acceptance of the MAA filed by Roche for Evrysdi for the treatment of SMA triggered a $15 million milestone payment to PTC and the first commercial sale of Evrysdi in the U.S. triggered a $20 million milestone payment to PTC. Additionally, in October 2020, Chugai Pharmaceutical, Co. Ltd, a member of the Roche group, announced that a new drug application for Evrysdi for the treatment of SMA was filed in Japan. The filing in Japan triggered a $7.5 million milestone payment to PTC in the fourth quarter of 2020. As a result, PTC recognized $35.0 million in the third quarter of 2020 and $7.5 million in the fourth quarter of 2020 of collaboration revenue associated with Roche milestone events.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $118 million for the fourth quarter of 2020, compared to $81.8 million for the fourth quarter of 2019. GAAP R&D expenses were $477.6 million for the full year 2020, compared to $257.4 million for the full year 2019. The increase in R&D expenses for the fourth quarter and full year 2020 reflects costs associated with advancing the gene therapy and Bio-e platforms, increased investment in research programs, and advancement of the clinical pipeline. Additionally, the increase in R&D expenses full year 2020 includes one-time charges in 2020 of $53.6 million related to the acquisition of Censa Pharmaceuticals, and $41.4 million related to the MassBiologics of the University of Massachusetts Medical School agreement for commercial manufacturing of our lead gene therapy program in AADC deficiency.
Non-GAAP R&D expenses were $105.3 million for the fourth quarter of 2020, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $76.2 million for the fourth quarter of 2019, excluding $5.6 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $438.9 million for the full year 2020, excluding $38.7 million in non-cash, stock-based compensation expense, compared to $236.6 million for the full year 2019, excluding $20.8 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $75.5 million for the fourth quarter of 2020, compared to $63.5 million for the fourth quarter of 2019. GAAP SG&A expenses were $245.2 million for the full year 2020, compared to $202.5 million for the full year 2019. The increase reflects continued investment to support PTC’s commercial activities including the expanding commercial portfolio, and rent and related expenses associated with entering into a long-term lease for the Hopewell facility that commenced on July 1, 2020.
Non-GAAP SG&A expenses were $66.8 million for the fourth quarter of 2020, excluding $8.7 million in non-cash, stock-based compensation expense, compared to $57.7 million for the fourth quarter of 2019, excluding $5.8 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $213.6 million for the full year 2020, excluding $31.6 million in non-cash, stock-based compensation expense, compared to $181.2 million for the full year 2019, excluding $21.3 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $6.3 million for the fourth quarter of 2020, compared to $12.4 million for the fourth quarter of 2019. Change in the fair value of deferred and contingent consideration was $23.3 million for the full year 2020, compared to $48.4 million for the full year 2019. The change is related to the fair valuation of the potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis), as a result of our merger with Agilis which closed in August 2018. Changes in the fair value were due to the re-calculation of discounted cash flows for the passage of time and changes to certain other estimated assumptions.
Settlement of deferred and contingent consideration was $10.6 million for the full year 2020. The settlement of deferred and contingent consideration is related to a loss upon the settlement of the deferred and contingent consideration liabilities as a result of the rights exchange agreement with certain former shareholders of Agilis, whereby such former shareholders exchanged their pro rata share of specific future cash milestone payments in the aggregate amount of $225 million for a mixture of cash and equity of PTC. Under this agreement, which the former shareholders and PTC entered into on April 29, 2020, PTC has paid $36.9 million in cash and issued 2,821,176 shares of common stock in exchange for the cancellation and forfeiture of the participating shareholders’ rights to receive (i) $174.0 million, in the aggregate, of potential milestone payments based on the achievement of certain regulatory milestones and (ii) $37.6 million, in the aggregate, of $40.0 million in development milestone payments that would have been due upon the passing of the second anniversary of the closing of PTC’s merger with Agilis, regardless of whether the milestones are achieved.
Net loss was $74.4 million for the fourth quarter of 2020, compared to net loss of $77.7 million for the fourth quarter of 2019. Net loss was $438.2 million for the full year 2020, compared to net loss of $251.6 million for the full year 2019.
Cash, cash equivalents, and marketable securities was $1.1 billion at December 31, 2020, compared to $686.6 million at December 31, 2019.
Shares issued and outstanding as of December 31, 2020 were 69,718,096.
PTC Reaffirms Full Year 2021 Guidance as Follows:

PTC anticipates net product revenues for the DMD franchise for the full year 2021 to be between $355 and $375 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $725 and $755 million, excluding estimated non-cash, stock-based compensation expense of $100 million.
Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 2174406. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. The accompanying slide presentation will be posted on the investor relations section of the PTC website. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On February 25, 2021 Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported financial results and provided business updates for the fourth quarter and fiscal year 2020 and highlighted pipeline progress (Press release, Moderna Therapeutics, FEB 25, 2021, View Source [SID1234575682]).

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"2020 was a historic year for Moderna. The team rose to the challenge to address the devastating COVID-19 pandemic in less than one year with our authorized vaccine. It is encouraging and humbling that more than 32 million doses of our vaccine have been administered in the U.S. and that millions of people around the world have been vaccinated with our vaccine to date. 2020 demonstrated the power of harnessing mRNA to make medicines and also demonstrated the speed and scalability of the Moderna platform that we have built over the last 10 years," said Stéphane Bancel, Chief Executive Officer of Moderna. "I believe that 2021 will be an inflection year for Moderna. We previously believed that mRNA would lead to approved medicines, and we were limited in our ambitions by the need for regular capital raises and keeping several years of cash to manage financing risk. We now know that mRNA vaccines can be highly efficacious and authorized for use, and we are a cash-flow generating commercial company. We opened commercial subsidiaries in 8 countries in 2020 and plan to add Japan, South Korea and Australia in 2021. We plan to accelerate and significantly increase our investments in science and grow our development pipeline faster. By executing on our 2021 priorities, we will advance our mission of delivering on the promise of mRNA science to create a new generation of transformative medicines for patients. This is just the beginning."

New updates and recent progress include:

COVID-19 Vaccine Development

Raises lower end of global manufacturing plan for 2021 from 600 million doses to 700 million doses; making capital investments to increase capacity up to an expected 1.4 billion doses in 2022
Phase 2/3 study of mRNA-1273 in adolescents has completed enrollment of 3,000 participants
Infectious Diseases

First cohort in the Phase 1 study of hMPV/PIV3 vaccine candidate (mRNA-1653) fully enrolled
First older adult in the Phase 1 study of RSV vaccine candidate (mRNA-1345) has been dosed
First 3 cohorts in age de-escalation study of RSV vaccine candidate (mRNA-1345) for the pediatric population fully enrolled
Rare Diseases

Study start-up activities for Phase 1/2 study of PA candidate (mRNA-3927) ongoing
Cardiovascular Diseases

Regained all rights to the Relaxin development candidate from AstraZeneca
Moderna currently has 24 mRNA development programs in its portfolio with 13 having entered clinical studies. The Company’s updated pipeline can be found at www.modernatx.com/pipeline. Moderna and collaborators have published more than 65 peer-reviewed papers.

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 governments and non-profit organizations.

COVID-19 Vaccine Development

Moderna COVID-19 Vaccine: On December 30, 2020, interim safety and primary efficacy results from the Phase 3 trial of the Moderna COVID-19 Vaccine (mRNA-1273) were published in the New England Journal of Medicine. Safety data continues to accrue, and the study continues to be monitored by an independent Data Safety Monitoring Board (DSMB) appointed by the National Institutes of Health (NIH). All participants in the COVE study will be monitored for two years after their second dose to assess long-term protection and safety. Today, the Company is sharing an update on the Phase 3 COVE study. An updated total of adjudicated COVID-19 cases among baseline seronegative participants in the COVE study starting two weeks following the second dose showed 690 cases to date, of which 639 cases of COVID-19 were observed in the placebo group versus 51 cases observed in the Moderna COVID-19 Vaccine group. Participants in the Phase 3 COVE study are in the process of being unblinded and cases will continue to accrue. On December 18, 2020, the U.S. Food and Drug Administration (FDA) authorized the emergency use of mRNA-1273, Moderna’s vaccine against COVID-19, in individuals 18 years of age and older. Moderna has also received emergency (or other conditional, interim or provisional) authorization for use of its COVID-19 vaccine from health agencies in Canada, Israel, the European Union, the United Kingdom, Switzerland, Singapore and Qatar. Moderna is working with additional health agencies and with the World Health Organization on the authorization of its vaccine in additional jurisdictions. BARDA, part of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), partially supported the research and development of the Moderna COVID-19 Vaccine with federal funding under Contract no. 75A50120C00034. Moderna retains worldwide rights to develop and commercialize the Moderna COVID-19 Vaccine.
Addressing Variants of Concern: On February 24, Moderna announced that it completed manufacturing of clinical trial material for its variant-specific vaccine candidate, mRNA-1273.351, against the SARS-CoV-2 variant known as B.1.351 first identified in the Republic of South Africa and has shipped doses to the NIH for a Phase 1 clinical trial that will be led and funded by the NIH’s NIAID. The Company also provided an update on its strategy for addressing SARS-CoV-2 variants of concern.
Publication of Note: Letter to the editor in the New England Journal of Medicine published February 17, 2021, showed vaccination with the Moderna COVID-19 Vaccine produced neutralizing titers against all key emerging variants tested, including B.1.1.7 and B.1.351, first identified in the UK and Republic of South Africa, respectively. The study showed no significant impact on neutralizing titers against the B.1.1.7 variant relative to prior variants. A six-fold reduction in neutralizing titers was observed with the B.1.351 variant relative to prior variants.
Further Clinical Studies of mRNA-1273
Phase 2/3 "TeenCOVE" study of mRNA-1273 in adolescents: The Phase 2/3 study of mRNA-1273 in adolescents ages 12-17 years has completed enrollment of 3,000 participants in the U.S.
Phase 2 "KidCOVE" study of mRNA-1273 in young children: The Phase 2 study of mRNA-1273 in pediatric population ages 6 months to 11 years will start in the near-term.
Phase 1/2 study of mRNA-1273 in Japan: The Phase 1/2 study of Moderna’s vaccine candidate against COVID-19 (mRNA-1273 or TAK-919) in Japan, led by Takeda Pharmaceutical Co., Ltd is ongoing.
Next-generation vaccine against COVID-19 (mRNA-1283): mRNA-1283 is a next-generation vaccine candidate against COVID-19 that encodes for the portions of the SARS-CoV-2 spike protein critical for neutralization, specifically the Receptor Binding Domain (RBD) and N-terminal Domain (NTD). The encoded mRNA-1283 antigen is shorter than mRNA-1273, and is being developed as a potential refrigerator stable mRNA vaccine that will facilitate easier distribution and administration by healthcare providers. mRNA-1283 is intended to be evaluated for use as a booster dose for previously vaccinated or infected individuals as well as in a primary series for seronegative individuals.
Vaccines requiring complex antigens and against highly prevalent infections

Cytomegalovirus (CMV) vaccine (mRNA-1647): Positive interim data from the Phase 2 study assessing the safety, reactogenicity, and immunogenicity of different dose levels of mRNA-1647 were presented at Moderna’s annual R&D Day. Based on the interim analysis of the Phase 2 study, the 100 μg dose has been chosen for the Phase 3 pivotal study, which is expected to begin in 2021. Moderna owns worldwide commercial rights for mRNA-1647.
Epstein-Barr virus (EBV) vaccine (mRNA-1189): mRNA-1189 is a vaccine against 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. Moderna is planning to begin a Phase 1 study of mRNA-1189 in 2021. There is no approved vaccine for EBV. Moderna owns worldwide commercial rights to mRNA-1189.
Vaccines against respiratory infections

Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3) vaccine (mRNA-1653): Moderna is enrolling seropositive pediatric participants (12-36 months of age) in the Phase 1 study of hMPV/PIV3 study (mRNA-1653). The first cohort in this study has been fully enrolled. Moderna owns worldwide commercial rights to mRNA-1653.
Respiratory syncytial virus (RSV) vaccine (mRNA-1345): mRNA-1345 is a vaccine against RSV encoding for a prefusion F glycoprotein, which elicits a superior neutralizing antibody response compared to the postfusion state. On January 11, Moderna announced its plan to amend the protocol to include evaluation of mRNA-1345 in older adults (greater than 50 years), in addition to the pediatric population, who are also at risk of significant RSV disease. In the pediatric population, the first three cohorts of younger adults in the Phase 1 age de-escalation study of mRNA-1345 are fully enrolled. The age range of toddlers in this de-escalation Phase 1 study has been amended to 12-59 months (from 12-36 months). In the older adult population, the first participant in the Phase 1 study of mRNA-1345 in the adult RSV vaccine has been dosed. The Company also intends to evaluate the potential of combinations of mRNA-1345 with its vaccines against other respiratory pathogens in children and separately in older adults. There is no approved vaccine for RSV. Moderna owns worldwide commercial rights to mRNA-1345.
Seasonal influenza vaccine (mRNA-1010, mRNA-1020, mRNA-1030): Seasonal flu (type A and type B) epidemics occur seasonally and vary in severity each year, causing respiratory illnesses and placing substantial burden on healthcare systems. The WHO estimates globally about 3,000,000-5,000,000 severe cases of flu each year, and 290,000-650,000 flu-related respiratory deaths. Approximately 8% of the U.S. population experiences symptoms from flu each year, with 140,000-810,000 hospitalizations and 12,000-61,000 deaths per year. Peak flu activity is seen in temperate climates from fall to winter and is reflected in increases in outpatient visits, urgent care visits, and hospitalizations. In the U.S., the estimated average economic burden of seasonal influenza is approximately $11 billion per year. The Company plans to explore potential combination vaccines against flu, SARS-CoV-2, RSV and human metapneumovirus (hMPV). The Company’s first-generation flu program will evaluate multiple candidates comprising multiple antigen combinations against the four seasonal viruses recommended by the WHO. The Company expects to begin Phase 1 clinical trials for this program in 2021.
Public health vaccines

Zika virus vaccine (mRNA-1893):Moderna is preparing for a Phase 2 study of mRNA-1893, which is expected to begin in 2021. mRNA-1893 is being developed in collaboration with BARDA. Moderna owns worldwide commercial rights to mRNA-1893.
HIV vaccine (mRNA-1644 & mRNA-1574): HIV is the virus responsible for acquired immunodeficiency syndrome (AIDS), a lifelong, progressive illness with no effective cure. Approximately 38 million people worldwide are currently living with HIV with 1.2 million in the U.S. Approximately 2 million new infections of HIV are acquired worldwide every year and approximately 690,000 people die annually due to complications from HIV/AIDS. The primary routes of transmission are sexual intercourse and IV drug use, putting young adults at the highest risk of infection. From 2000 to 2015, a total of $562.6 billion globally was spent on care, treatment and prevention of HIV, representing a significant economic burden. mRNA-1644, a collaboration with the International AIDS Vaccine Initiative (IAVI) and the Bill and Melinda Gates Foundation (BMGF), is a novel approach to HIV vaccine strategy in humans designed to elicit broadly Neutralizing HIV-1 Antibodies (bNAbs). A Phase 1 study for mRNA-1644 will use iterative human testing to validate the approach and antigens and multiple novel antigens will be used for germline-targeting and immuno-focusing. A second approach, mRNA-1574, is being evaluated in collaboration with the NIH and includes multiple native-like trimer antigens. The Company expects to begin Phase 1 studies for both mRNA-1644 and mRNA-1574 in 2021.
Nipah virus (NiV) Vaccine (mRNA-1215): NiV is a zoonotic virus transmitted to humans from animals, contaminated food, or through direct human-to-human transmission and causes a range of illnesses including fatal encephalitis. Severe respiratory and neurologic complications of NiV have no treatment other than intensive supportive care. The case fatality rate among those infected is estimated at 40-75%. NiV outbreaks cause significant economic burden to impacted regions due to loss of human life and interventions to prevent further spread, such as the slaughter of infected animals. NiV has been identified as the cause of isolated outbreaks in India, Bangladesh, Malaysia, and Singapore since 2000 and is included on the WHO R&D Blueprint list of epidemic threats needing urgent R&D action. mRNA-1215 was co-developed by Moderna and the NIH’s Vaccine Research Center (VRC).
Pandemic influenza/H7N9 vaccine (mRNA-1851): Discussions regarding funding the Company’s pandemic influenza/H7N9 vaccine program through approval are ongoing.
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): Positive interim data from the Phase 1 study evaluating escalating doses of mRNA-1944 in the 0.6 mg/kg dose with steroid premedication cohort and two doses of 0.3 mg/kg (without steroid premedication) given one week apart cohort were presented at Moderna’s annual R&D Day in September and demonstrated dose-dependent increases in levels of antibody against chikungunya. Safety and increased CHKV-IgG production in the two-dose regimen shows the platform’s ability for repeat dosing.
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): Moderna has regained all rights to the Relaxin development candidate from AstraZeneca. Moderna now owns worldwide commercial rights to this development candidate.
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. Phase 1 in multiple cohorts is ongoing. The upsized head & neck cohort is recruiting additional patients. Moderna shares worldwide commercial rights to mRNA-4157 with Merck.
Mutant 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 Phase 1/2 study of mRNA-2416 alone and in combination with durvalumab (IMFINZI) is ongoing. The Phase 2 dose expansion study of mRNA-2416 in combination with durvalumab in ovarian cancer patients is enrolling and the first patients have been dosed. 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 VEGF-A, which is being developed for patients with ischemic heart disease 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.

Propionic acidemia (PA) (mRNA-3927): Study start-up activities for the Phase 1/2 study of PA candidate (mRNA-3927) have resumed following COVID-19 related pause and protocol amendment. mRNA-3927 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-3927.
Methylmalonic acidemia (MMA) (mRNA-3705): Moderna received rare pediatric designation for its next generation MMA candidate (mRNA-3705). The Company plans to file new IND and CTA applications for mRNA-3705 and will focus development efforts on that candidate going forward. mRNA-3705 uses the same LNP formulation as mRNA-1944. Moderna owns worldwide commercial rights to mRNA-3705.
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 the 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 can be found on the investor relations page of its website: investors.modernatx.com.

Fourth Quarter and Full Year 2020 Financial Results (Unaudited)

Cash Position: Cash, cash equivalents and investments as of December 31, 2020 and 2019 were $5.25 billion and $1.26 billion, respectively.
Net Cash Provided by (Used in) Operating Activities: Net cash provided by operating activities was $2.03 billion for the year ended December 31, 2020 compared to net cash used in operating activities of $(459) million for the year ended December 31, 2019.
Cash Used for Purchases of Property and Equipment: Cash used for purchases of property and equipment was $67 million for the year ended December 31, 2020 compared to $32 million for the same period in 2019.
Revenue: Total revenue was $571 million for the fourth quarter of 2020 compared to $14 million for the fourth quarter of 2019. Total revenue was $803 million for the year ended December 31, 2020 compared to $60 million for the year ended December 31, 2019. The increases in both periods in 2020 were driven by increases in grant revenue and product sales. The increase in grant revenue was primarily due to the BARDA award to accelerate development of our COVID-19 vaccine. We began to recognize revenue in December 2020 from our COVID-19 vaccine subsequent to its authorization for emergency use by the FDA and Health Canada.
Cost of Sales: Costs of sales were $8 million, or 4% of Moderna’s product sales, in 2020, primarily comprised of third-party royalties as the associated inventory costs were expensed previously. If inventory sold during 2020 was valued at cost, Moderna’s cost of sales for 2020 would have been $62 million, or 31% of Moderna’s product sales.
Research and Development Expenses: Research and development expenses were $759 million for the fourth quarter of 2020 compared to $118 million for the fourth quarter of 2019. Research and development expenses were $1.37 billion for the year ended December 31, 2020 compared to $496 million for the year ended December 31, 2019. The increases in 2020 were largely attributable to mRNA-1273 clinical development, pre-launch inventory buildup prior to the emergency use authorization from the FDA and to a lesser extent, an increase in personnel related costs, primarily driven by an increase in the number of employees supporting our mRNA-1273 development activities.
Selling, General and Administrative Expenses: Selling, general and administrative expenses were $79 million for the fourth quarter of 2020 compared to $26 million for the fourth quarter of 2019. Selling, general and administrative expenses were $188 million for the year ended December 31, 2020 compared to $110 million for the year ended December 31, 2019. The increases in 2020 were mainly due to an increase in personnel costs, and outside services, primarily attributable to increased headcount and mRNA-1273 vaccine candidate commercialization-related activities.
Net Loss: Net loss was $272 million for the fourth quarter of 2020 compared to $123 million for the fourth quarter of 2019. Net loss was $747 million for the year ended December 31, 2020 compared to $514 million for the year ended December 31, 2019.
Moderna’s COVID-19 Vaccine Supply Agreements & Regulatory Updates

Moderna has confirmed the following supply agreements of committed orders:

United States: 300 million doses with options to purchase an additional 200 million doses; the U.S. Food and Drug Administration (FDA) has authorized emergency use in individuals 18 years of age and older
European Union: 310 million doses with option to purchase an additional 150 million doses in 2022; the European Commission has granted a conditional marketing authorization (CMA) for COVID-19 Vaccine Moderna in individuals 18 years of age and older1
Japan: 50 million doses
Canada: 44 million doses; Health Canada authorized COVID-19 Vaccine Moderna for the immunization of people 18 years of age and older under an Interim Order
Republic of Korea: 40 million doses
United Kingdom: 17 million doses; the UK Medicines and Healthcare products Regulatory Agency (MHRA) approved the COVID-19 Vaccine Moderna for use under Regulation 174, a temporary authorization
Switzerland: 13.5 million doses; Swissmedic, the Swiss Agency for Therapeutic Products, authorized the COVID-19 Vaccine Moderna in Switzerland
Colombia: 10 million doses
Israel: 6 million doses; Israel’s Ministry of Health (MOH) has given authorization to import the COVID-19 Vaccine Moderna
Taiwan: 5 million doses
Singapore: (undisclosed); the Singapore Health Sciences Authority (HSA) approved the interim authorization of the COVID-19 Vaccine Moderna for use under the Pandemic Special Access Route (PSAR)
Qatar (undisclosed); the Qatar Ministry of Public Health issued an emergency use authorization for the COVID-19 Vaccine Moderna
Management Updates

Moderna’s Chief Medical Officer (CMO), Tal Zaks, M.D., Ph.D., will be leaving the Company in late September after six years of service. The Company has retained Russell Reynolds to recruit for a new CMO with global and commercial experience as the Company scales up the launch of its COVID-19 Vaccine and prepares to file several biologics license applications (BLAs) over the next few years.
"I would like to thank Tal for his tremendous impact on Moderna’s success over the last six years. Tal joined us when we were a pre-clinical company. His guidance and contributions were important in helping Moderna get to where we are today. Through his leadership over the past year in Moderna’s response to the COVID-19 pandemic, Tal has made a contribution that extends beyond Moderna to all of society. I have enjoyed having him as my partner and wish him all the best as he embarks on the next leg of his career," said Stéphane Bancel.

Corinne Le Goff, Pharm.D., M.B.A., joined Moderna as the Company’s first Chief Commercial Officer on January 19, 2021. Dr. Le Goff previously served as SVP and President U.S. Business Organization at Amgen (Nasdaq: AMGN). During her nearly 6-year tenure at Amgen, she also served as SVP of the Europe Region and oversaw 48 markets. Dr. Le Goff was actively engaged with the policy community and advocates for innovative, high-quality and affordable healthcare.
2020 Commercial Network

The Company started to build a global commercial network. This infrastructure will enable Moderna’s entire portfolio, which means that Moderna can commercialize its entire pipeline without a large pharmaceutical partner. The Company now has subsidiaries, distributors or partners in the following geographies:

Moderna USA
Moderna Canada
Moderna France
Moderna Germany
Moderna Italy
Japan (Partner: Takeda)
Moderna Spain
Moderna UK
Moderna Switzerland
Corporate Updates

Shareholder letter: Moderna CEO Stéphane Bancel published a letter to shareholders on January 4, 2021.
Annual Meeting of Shareholders: The Moderna Annual Meeting of Shareholders will be held on April 28, 2021 at 8:00 a.m. ET. The meeting will be held virtually at www.virtualshareholdermeeting.com/MRNA2021. The record date for voting or attendance as a stockholder is 4:00 p.m. ET on March 1, 2021.
2021 Financial Considerations

Advance Purchase Agreements (APAs): Already signed APAs for scheduled delivery in 2021, reflecting a total of $18.4 billion in anticipated product sales. Additional discussions ongoing with several governments relating to APAs for scheduled deliveries in 2021 and 2022. Moderna responded to a tender to UNICEF to supply COVAX in 2021 and 2022 and discussions are ongoing with COVAX/UNICEF.
Cost of Sales: Expected at approximately 20% of product sales for fiscal year 2021.
2021 Research & Development (R&D) and Selling, General & Administrative (SG&A) Expenses: For the first quarter 2021, expect low double-digit percent increase versus adjusted fourth quarter 2020 results of $0.5 billion. The fourth quarter 2020 reported expense was $0.8 billion. Adjusted Q4 expense, totaling $0.5 billion, excludes $0.3 billion of reported expenses, which are now capitalized and expensed through cost of sales, based on our change from a research and development to a commercial organization.
Tax Rate: Effective tax rate expected in the mid-teen percentage level as a result of the forecasted global sales mix and utilization of the accumulated net operating loss carry-forward of $2.3 billion.
Capital Expenditures: $350-400 million of capital investments currently planned for 2021.
2021 New Commercial Networks

In 2021, the Company plans to further expand its commercial network to enable Moderna’s entire portfolio.

Moderna will host a live conference call and webcast at 8:00 a.m. ET on Thursday, February 25, 2021. To access the live conference call, please dial 866-922-5184 (domestic) or 409-937-8950 (international) and refer to conference ID 4066945. 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 one year following the call.

On February 25, 2021 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need, reported operational highlights and financial results for the year ended December 31, 2020 on Thursday, March 4, 2021, and will host a conference call the same day at 4:30 p.m. ET (Press release, Synthetic Biologics, FEB 25, 2021, View Source [SID1234575720]). The dial-in information for the call is as follows:

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!

Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source . An archived replay of the call will be available for approximately ninety (90) days at the same URL, View Source beginning approximately one hour after the call’s conclusion.

On February 25, 2021 LIDDS reported that Interim report January – December 2020 (Press release, Lidds, FEB 25, 2021, View Source [SID1234575741])

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!

JANUARY – DECEMBER 2020

Net sales amounted to MSEK 0.3 (0.0)
Operating expenses amounted to MSEK -33.0 (-31.4)
Profit/loss before and after tax amounted to MSEK -32.3 (-31.4)
Earnings per share amounted to SEK -1.20 (-1.34)
Cash flow from operating activities amounted to MSEK -27.4 (-31.2)
Equity amounted to MSEK 42.8 (15.5) and the debt/equity ratio was 79% (72%)
OCTOBER – DECEMBER 2020

Net sales amounted to MSEK 0.3 (0.0)
Operating expenses amounted to MSEK -12.9 (-8.5)
Profit/loss before and after tax amounted to MSEK -12.5 (-8.5)
Earnings per share amounted to SEK -0.42 (-0.36)
Cash flow from operating activities amounted to MSEK -10.1 (-9.8)
Equity amounted to MSEK 42.8 (15.5) and the debt/equity ratio was 79% (72%)
SIGNIFICANT EVENTS DURING THE FORTH QUARTER 2020

Intratumoral injection of NZ-TLR9, NanoZolid formulation of a Toll-Like Receptor 9 (TLR9), results in strong antitumoral efficacy combined with prominent antitumoral immune responses in mouse tumor models. NZ-TLR9 forms an intratumoral depot which releases the TLR9 agonist for least 6 weeks and thus minimizes the need for repeated injections.
The warrants programme was finalized in October 2020 and 0.8 percent of the warrants issued was exercised.
A newly revised guideline from the Chinese National Medical Products Administration (NMPA) has extended the requirements to a full registration dossier for the conditional market approval (CMA). LIDDS licensee Jiangxi Puheng Pharma is therefore aiming to submit the application for CMA for the prostate cancer drug candidate Liproca Depot in China during Q1, 2021.
A review article in OncoTargets and Therapy journal defines LIDDS as a key player in TLR9 agonist research field and being the only provider having a sustained release product in development.
LIDDS announced that the company will apply for a relisting of its shares from Nasdaq First North to Nasdaq Stockholm Main Market in 2021.
Nina Herne will assume the role of CEO for LIDDS as from April 19, 2021. Monica Wallter will continue in LIDDS as Senior Adviser.
SIGNIFICANT EVENTS FOLLOWING THE END OF THE PERIOD

The Phase I study where NanoZolid is combined with docetaxel continues with dose escalation. New patients are in recruitment to receive injection in solid tumors with new dose of docetaxel.
European Urology Focus has accepted a scientific article describing LIDDS Phase IIb study results with Liproca Depot.
LIDDS has filed a patent application for local intracranial treatment of brain tumors. The NanoZolid technology enables controlled and sustained release of oncology drugs. Preclinical study has showed that NanoZolid injected as a depot in brain is tolerable and safe.
LIDDS has signed a manufacturing agreement for NZ-TLR9 with Pharmidea in Latvia.

The interim report is available on the company’s website, go to View Source

On February 25, 2021 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose of bettering the lives of people impacted by kidney disease, reported financial results for the fourth quarter and full-year ended December 31, 2020 and provided business updates (Press release, Akebia, FEB 25, 2021, View Source [SID1234575585]). The Company will host a conference call today, Thursday, February 25, 2021, at 9:00 a.m. Eastern Time.

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!

Akebia also announced a $60 million non-dilutive transaction with an entity managed by HealthCare Royalty Management, LLC (HCR), to monetize the Company’s rights to receive royalties and sales milestones on vadadustat net sales under its collaboration agreement with Mitsubishi Tanabe Pharma Corporation (MTPC). MTPC has the exclusive rights to commercialize vadadustat in Japan, where it is currently marketed under the trade name Vafseo (vadadustat), and certain other Asian countries. Under the terms of the agreement with HCR, Akebia receives an upfront cash payment of $45 million and is eligible to receive an additional $15 million if certain sales milestones are achieved. In exchange, HCR has the right to receive Vafseo royalties and sales milestones due to the Company under its collaboration agreement with MTPC, subject to an annual cap of $13 million and an aggregate cap of $150 million. After the annual cap is met in a given calendar year, Akebia will recognize 85 percent of Vafseo royalties and sales milestones from MTPC for that year. After the aggregate cap is met, Akebia will recognize 100 percent of Vafseo royalties and sales milestones until this revenue stream ends under the terms of the Company’s collaboration agreement with MTPC. The transaction does not include potential future regulatory milestones to be paid by MTPC.

"2020 was a year of focused execution for Akebia as we advanced vadadustat, our investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), and executed on our commercial priorities. Importantly, we achieved this while continuing to provide patients with access to our therapies and keeping with our goal of maintaining a strong balance sheet," stated John P. Butler, Chief Executive Officer of Akebia. "With this forward momentum, we have line of sight to significant milestones in 2021, including our NDA submission for vadadustat for the treatment of anemia due to chronic kidney disease (CKD) in both adult patients on dialysis and not on dialysis expected by the middle of the second quarter of this year."

Butler continued, "As we advance vadadustat toward commercialization, subject to approval, we continue to execute on key commercial, development, and financial priorities. As dialysis is a primary focus, we’re thrilled to have recently added LeAnne Zumwalt to our Board of Directors. LeAnne’s perspective and extensive operating experience with one of the largest dialysis operators in the U.S. will help ensure that our market and commercial strategies are well aligned with the needs of dialysis providers and their patients. We’re also pleased to have announced a $60 million royalty monetization transaction with HCR that we believe strengthens our balance sheet and helps preserve both our strategic and financial flexibility while we continue investing for the successful launch of vadadustat, upon approval."

Akebia plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for vadadustat by the middle of the second quarter of this year for the treatment of anemia due to CKD in both adult patients on dialysis and adult patients not on dialysis. In addition, Akebia and its collaborator, Otsuka Pharmaceutical Co. Ltd. (Otsuka), are working in close collaboration to prepare a Marketing Authorization Application (MAA) for submission to the European Medicines Agency (EMA), expected this year.

Recent Business Highlights:

In February, the Company announced that LeAnne M. Zumwalt joined Akebia’s Board of Directors. Ms. Zumwalt recently served as Group Vice President, Government Affairs at DaVita Inc.
In February, Akebia launched its Medical Engagement Hub, an online resource dedicated to scientific education and connecting U.S. healthcare professionals with Akebia Medical Affairs.
In January, the University of Texas Health Science Center at Houston (UTHealth) in Houston, Texas, announced that it had received $5.1 million in government funding for its study evaluating the use of vadadustat as a potential therapy to prevent and lessen the severity of acute respiratory distress syndrome (ARDS), a complication of COVID-19. This investigator-sponsored research study is currently underway and actively enrolling patients.
In November, the study design and methodology for Akebia’s global Phase 3 INNO2VATE program was published in Nephrology Dialysis Transplantation (NDT).
In October, the study design and methodology for Akebia’s global Phase 3 PRO2TECT program was published in American Heart Journal.
In October, Akebia completed a pre-NDA meeting with the FDA for vadadustat.
In October, top-line data from both INNO2VATE and PRO2TECT were presented at American Society of Nephrology Kidney Week 2020 Reimagined.
In October, Akebia and Otsuka launched Balancing Anemia Due to CKD, a campaign and website designed to increase awareness and education of anemia due to CKD among healthcare providers with the goal of improving the management of this disease for patients.
Fourth Quarter and Full-Year 2020 Financial Results

Revenues: Total revenue was $56.7 million for the fourth quarter of 2020 compared to $69.6 million for the fourth quarter of 2019, and $295.3 million for the full-year 2020 compared to $335.0 million for the full-year 2019. The decline in both periods was driven by lower collaboration revenue consistent with the Company successfully completing its global Phase 3 clinical development program for vadadustat, which consisted of two programs that evaluated the efficacy and safety of vadadustat versus darbepoetin alfa for the treatment of anemia due to CKD in adult patients on dialysis (INNO2VATE) and not on dialysis (PRO2TECT).
Collaboration revenue was $22.1 million for the fourth quarter of 2020 compared to $40.6 million for the fourth quarter of 2019, and $166.4 million for the full-year 2020 compared with $223.9 million for the full-year 2019. The change in both periods is due to the timing of when vadadustat development expenses are incurred and the associated revenue is recognized on a percentage-of-completion basis. Collaboration revenue for the full-year 2020 includes $0.4 million in royalty revenue related to the commercial sale of Vafseo in Japan from MTPC, which launched in August of 2020.
Net product revenue was $34.6 million for the fourth quarter of 2020 compared with $28.9 million for the fourth quarter of 2019, an increase of 20 percent. Net product revenue was $128.9 million for the full-year 2020 compared to $111.1 million for the full-year 2019, an increase of 16 percent. While Akebia did not experience a significant impact from COVID-19 on revenues during the first nine months of 2020, the Company believes its revenue growth was negatively impacted during the fourth quarter of 2020 primarily as the kidney patient populations that it serves continue to experience both higher hospitalization and mortality rates due to COVID-19.
COGS: Cost of goods sold was $63.2 million for the fourth quarter of 2020 compared to $38.1 million for the fourth quarter of 2019. Excluding non-cash purchase accounting adjustments as a result of the merger with Keryx, the increase in the fourth quarter of 2020 was primarily driven by a $14.8 million non-cash charge related to excess purchase commitments. Cost of goods sold was $295.9 million for the full-year 2020, compared with $145.3 million for the full-year 2019. Cost of goods sold for 2020 includes a $115.5 million non-cash charge related to the impairment of the Auryxia intangible asset in the second quarter of 2020, a $25.1 million non-cash charge related to excess purchase commitments, and a $20.1 million non-cash charge for inventory write-downs largely related to a previously disclosed manufacturing quality issue related to Auryxia.
R&D Expenses: Research and development expenses were $37.6 million for the fourth quarter of 2020 compared to $80.4 million for the fourth quarter of 2019, and $218.5 million for the full-year 2020 compared to $323.0 million for the full-year 2019. The decline in both periods was primarily driven by a decrease in costs consistent with the Company completing the INNO2VATE and PRO2TECT studies.
SG&A Expenses: Selling, general and administrative expenses were $40.3 million for the fourth quarter of 2020 compared to $44.9 million for the fourth quarter of 2019, and $153.9 million for the full-year 2020 compared to $149.5 million for the full-year 2019.
Net Loss: Net loss was $87.0 million for the fourth quarter of 2020 compared to $94.5 million for the fourth quarter of 2019, and $383.5 million for the full-year 2020 compared to $279.7 million for the full-year 2019. The increase in net loss for the full-year 2020 compared to the prior year period was due primarily to lower collaboration revenue and higher cost of goods sold, partially offset by lower operating expenses.
Cash Position: Cash, cash equivalents and available-for-sale securities as of December 31, 2020 were $268.7 million. The Company expects its cash resources to fund its current operating plan beyond the expected U.S. launch of vadadustat, assuming timely regulatory approval and the receipt of associated regulatory milestones.
"As COVID-19 continues to adversely and disproportionately impact our patient population with higher hospitalization and mortality rates, we expect this will have a negative impact on future Auryxia revenue growth. While we are unable to fully quantify the impact of the COVID-19 pandemic on future revenues and revenue growth, we continue to work to position the Company to navigate these challenges. As such, our financial priorities remain focused on improving our cost structure and maintaining a strong balance sheet, as evidenced by our recent $60 million non-dilutive royalty transaction with HCR," stated David A. Spellman, Chief Financial Officer of Akebia Therapeutics.

Conference Call
Akebia will host a conference call at 9:00 a.m. Eastern Time today, Thursday, February 25th, to discuss its fourth quarter and full-year 2020 financial results and recent business highlights. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 5455117. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through March 3, 2021. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 5455117. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source