On February 27, 2020 Lipocine Inc. (NASDAQ:LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported the closing of a registered direct offering of 10,084,034 Class A Units, each consisting of one share of its common stock and one half of a common warrant to purchase one share of its common stock, at a price of $0.595 per Class A Unit, for total gross proceeds to the Company of approximately $6 million, before deducting placement agent fees and other estimated offering expenses (Press release, Lipocine, FEB 27, 2020, View Source [SID1234554936]). The Company intends to use the net proceeds from this offering for working capital and general corporate purposes.

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Roth Capital Partners acted as sole agent for the offering.

The offering was made pursuant to a shelf registration statement on Form S-3 (File No. 333-220942) (including a prospectus) previously filed with and declared effective by the U.S. Securities and Exchange Commission (the "SEC"). A prospectus supplement describing the terms of the offering has been filed with the SEC.

A copy of the prospectus supplement and the accompanying prospectus relating to these securities may be obtained by contacting Roth Capital Partners, 888 San Clemente Drive, Suite 400, Newport Beach, CA 92660, (800) 678-9147. Electronic copies of the prospectus supplement and the accompanying prospectus are also available free of charge on the website of the SEC at www.sec.gov.

This press release does not constitute an offer to sell or the solicitation of an offer to buy securities, nor will there be any sale of the securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

On February 27, 2020 BioInvent reported that Financial Statement January 1 – December 31, 2019 (Press release, BioInvent, FEB 27, 2020, View Source;december-31-2019-301012311.html [SID1234554953]).

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"We are proud that we have delivered on our goals for the year and feel confident that we will continue to do so in 2020, with a number of important milestones approaching. Our cooperation with Merck & Co is our second large pharma collaboration and an excellent validation of BioInvent’s work, as we develop BI-1206 in solid tumors as well as in haematological cancers."

Martin Welschof, CEO BioInvent

Financial information

Fourth quarter 2019

Net sales SEK 25.4 (10.4) million.
Loss after tax SEK -40.9 (-32.7) million.
Loss after tax per share before and after dilution SEK -0.08 (-0.09).
Cash flow from operating activities and investment activities SEK -28.5 (-38.2) million.
January – December, 2019

Net sales SEK 93.7 (38.5) million.
Loss after tax SEK -138.6 (-123.2) million.
Loss after tax per share before and after dilution SEK -0.31 (-0.36).
Cash flow from operating activities and investment activities SEK -129.3 (-145.2) million. Liquid funds as of December 31, 2019: SEK 154.0 (68.9) million.
Events in the fourth quarter

BioInvent entered into a clinical trial collaboration and supply agreement with Merck & Co to evaluate BI-1206 in combination with KEYTRUDA in advanced solid tumors. (R)
Selection of second target and extension of the research collaboration and license agreement with Pfizer Inc. announced. (R)
BioInvent and Transgene announced compelling preclinical data for BT-001 in solid tumors.
Manufacturing agreement signed with Cancer Research UK expected to generate approximately SEK 30 million (~$3 million). (R)
BI-1206 preclinical data in mantle cell lymphoma presented at ASH (Free ASH Whitepaper) 2019.
(R)= Regulatory event
Comments from the CEO

As we look back at 2019 for BioInvent, we can be proud of the delivery on our goals for the year. It is particularly exciting to pursue our lead clinical candidate BI-1206 in solid tumors as well as in hematological cancers.

In December, we concluded an agreement with Merck & Co. to evaluate the combination of BI-1206 and Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in a Phase l/lla clinical trial for the treatment of advanced solid tumors. This expands BI-1206 clinical development and builds on preclinical data that demonstrates its ability to address an important mechanism of resistance to PD-1 inhibition, in combination, with one of the most successful immune-oncology drug. The collaboration is an excellent validation of our work and scientific excellence, as Merck has carefully evaluated the pre-clinical data and mode of action that Bioinvent has generated. They have provided insightful feedback on the clinical protocol, and provided input on the strategy for the development of BI-1206 before concluding this agreement.

Our partnership with Pfizer is also progressing well, and further validates the high scientific quality of the work performed by the team at BioInvent. Pfizer has now selected the second target under our cancer immunotherapy research collaboration and license agreement, and we have extended the research term by six months.

These agreements with two of the largest and most highly-respected pharmaceutical companies in the world strongly endorse BioInvent’s proprietary F.I.R.S.T platform. The platform enables us to simultaneously identify targets and high-quality antibodies that bind to them and generates promising new drug candidates that broaden our pipeline and create licensing and partnering opportunities.

Importantly, we also presented pre-clinical data indicating a broad and clinically relevant role of FcγRIIb in mantle cell lymphoma and highlight the potential of BI-1206 to help overcome resistance to treatment in this disease. This further reinforces our belief that FcγRIIb will become a key component for the treatment of advanced hematological and solid malignancies.

We have made important strategic advances in our collaboration with Cancer Research UK (CRUK). Given the overlap with BioInvent’s own Phase I/IIa trial of BI-1206 in combination with rituximab in Non-Hodgkin Lymphoma (NHL), and the fact that standard of care for patients with chronic lymphocytic leukemia (CLL) has dramatically evolved over the last few years, recruitment in the UK study has become increasingly challenging – in particular since CRUK can only carry out trials in the UK. For these reasons we have agreed to limit the CRUK study to monotherapy, which is almost completed. This will result in a more complementary work and more efficient use of resources. BioInvent and CRUK look forward to explore the possibilities for a continued collaboration as we move forward.

We reported compelling results from extensive in vitro and in vivo preclinical studies with BT-001, an oncolytic virus expressing our proprietary anti-CTLA4 antibody and the cytokine GM-CSF. We are developing BT-001 in collaboration with Transgene and intend to submit a clinical trial application in Q1 2020. Preclinical data on BT-001 will be presented at scientific meetings in the coming months.

We anticipate several important milestones in 2020. This will include early results from the Phase I open label study with a combination of BI-1206 and rituximab in indolent NHL in the second half of the year. We will also be initiating the Phase l/lla study of BI-1206 in combination with pembrolizumab, as mentioned above, with early results from the Phase I study expected in the second half of 2021. We are expecting to advance two compounds into clinical programs in solid cancer: in 2020 the anti-TNFR2 antibody BI-1808, as single agent and in combination with an anti-PD1 antibody; and in 2021 the anti-FcγRllB antibody BI-1607 in combination with a checkpoint inhibitor.

As BioInvent continues to bring new opportunities and programs towards clinical development, financing is of course, a focus and priority for us, and we will continue to use a combination of sources for funding. Firstly, we are engaged in several business development discussions with the aim of partnering one or more of the programs in our portfolio. Secondly, the collaboration with Pfizer, which is also a model for other potential collaborations which commercialize our platform. Thirdly, our manufacturing capabilities generate revenue, with the most recent agreement with CRUK expected to generate SEK 30 million. CRUK has the potential to become a long-term strategic partner, as it works with a number of small- to mid-sized companies that need manufacturing support. And our fourth option is to use capital markets for financing. Based on the support from our large institutional investors and increased interest in our programs we feel optimistic that a combination of these four sources will continue to support BioInvent financially.

BioInvent consistently delivered on its strategy in 2019 and this is continuing into 2020. We are looking forward to keeping you updated on the exciting developments ahead.

On February 26, 2020 OPKO Health, Inc. (NASDAQ: OPK) reported business highlights and financial results for the three months ended December 31, 2019 (Press release, Opko Health, FEB 26, 2020, View Source [SID1234554799]).

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Business Highlights

Successful Phase 3 results for Somatrogon, an investigational long-acting human growth hormone to treat children with growth hormone deficiency: On October 21, 2019, OPKO and Pfizer announced the global Phase 3 trial evaluating somatrogon dosed once-weekly in pre-pubertal children with growth hormone deficiency (GHD) met its primary endpoint of non-inferiority to daily GENOTROPIN (somatropin) for injection, as measured by annual height velocity at 12 months.

Somatrogon Phase 3 clinical trial data to be presented at ENDO on March 28, 2020: Two oral presentations will be delivered at the Endocrine Society’s ENDO 2020 meeting, to be held March 28-31, 2020:

"Somatrogon Growth Hormone in the Treatment of Pediatric Growth Hormone Deficiency: Results of the Pivotal Phase 3" and

"Interpretation of Insulin-like Growth Factor (IGF-1) Levels Following Administration of Somatrogon (a long-acting Growth Hormone-hGH-CTP)"

Somatrogon regulatory submissions: Regulatory submission in the U.S. is anticipated in 2H 2020: Europe is expected to follow upon completion of the open-label study demonstrating benefit and compliance with reduced treatment burden, which is expected to be completed in the third quarter of 2020. The registration study in pediatric GHD patients in Japan is on track for completion in the first quarter of 2020.

RAYALDEE total prescriptions reported by IQVIA increased 89% in 4Q 2019 compared with 4Q 2018: Total prescriptions for the three months ended December 31, 2019 increased to more than 17,700, compared with approximately 9,400 during the comparable period of 2018.

RAYALDEE Phase 2 trial: Interim readout is expected in 1Q 2020 of the open-label Phase 2 trial with RAYALDEE in adults with vitamin D insufficiency and stage 5 chronic kidney disease (CKD) requiring dialysis.

4Kscore test receives final Local Coverage Determination for Medicare payments: On November 15, 2019, OPKO announced that Novitas Solutions issued its final Local Coverage Determination for Medicare payments for the 4Kscore test with defined coverage criteria, effective

December 30, 2019. 4Kscore utilization remained strong with nearly 18,000 tests performed during the fourth quarter of 2019. A premarket approval submission for the 4Kscore test was accepted for review by the U.S. Food and Drug Administration (FDA) in December 2019.

Financial Highlights

Consolidated revenues for the fourth quarter of 2019 increased to $224.3 million compared with $221.9 million for the comparable period of 2018. Revenue from services in the fourth quarter of 2019 was $177.9 million, revenue from products was $32.0 million including RAYALDEE net revenue of $12.6 million, and revenue from licensing and intellectual property was $14.4 million.

Operating expenses for the fourth quarter of 2019 were $336.8 million compared with $311.9 million during the fourth quarter of 2018. Operating expenses during the three months ended December 31, 2019 included a non-cash impairment charge of $91.8 million for goodwill and intangible assets related to the acquisitions of Claros, CURNA and Transition Therapeutics, while the 2018 period included a $21.8 million non-cash impairment charge. Selling, general and administrative expenses decreased during the 2019 period by $16 million to $79.1 million, while research and development expenses decreased to $23.0 million from $33.3 million for the 2018 period, primarily due to the completion of the pediatric Phase 3 study for OPKO’s long-acting human growth hormone product and other ongoing clinical trials.

The net loss for the fourth quarter of 2019, which includes a non-cash impairment charge of $91.8 million, was $112.4 million, or $0.18 per share, compared with a net loss of $76.1 million, or $0.13 per share, for the comparable period of 2018. The 2018 reported net loss benefited from inclusion of an income tax benefit of $28.3 million whereas the 2019 period included an income tax expense of $3.4 million.

Cash, cash equivalents and marketable securities were $85.5 million as of December 31, 2019. In the fourth quarter of 2019, the Company raised gross proceeds of $81.3 million from an underwritten public offering of common stock. On February 25, 2020, the Company entered into an unsecured credit facility providing $100 million of incremental capital on a non-dilutive basis. The credit facility is with Phillip Frost, MD, the Company’s Chairman and CEO.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today, Wednesday, February 26, 2020. The conference call dial-in and webcast information is as follows:
DOMESTIC DIAL-IN:

(877) 783-8475
INTERNATIONAL DIAL-IN:

(614) 999-1827
PASSCODE:

9678678
WEBCAST:

OPKO 4Q19 Results Conference Call

For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 9678678. The replay can be accessed for a period of time on OPKO’s website at OPKO 4Q19 Results Conference Call.

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 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the company will webcast its corporate presentations at the following investor conferences (Press release, Jazz Pharmaceuticals, FEB 26, 2020, View Source [SID1234554831]):

Schedule your 30 min Free 1stOncology Demo!
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                  Schedule Your 30 min Free Demo!

Cowen 40th Annual Healthcare Conference in Boston, MA on Tuesday, March 3, 2020 at 8:00 a.m. EST / 1:00 p.m. GMT. Bruce Cozadd, chairman and chief executive officer, will provide an overview of the company and a business and financial update.
Barclays Global Healthcare Conference in Miami Beach, FL on Tuesday, March 10, 2020 at 9:30 a.m. EDT / 1:30 p.m. GMT. Daniel Swisher, president and chief operating officer, will provide an overview of the company and a business and financial update.
A live audio webcast of each presentation may be accessed from the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of each presentation to ensure adequate time for any software downloads that may be necessary to listen to the webcast.

An archive of each webcast will be available for at least one week following each presentation on the Investors section of the company’s website at www.jazzpharmaceuticals.com.