On March 5, 2020 DermTech, Inc. (NASDAQ:DMTK) ("DermTech"), a leader in precision dermatology enabled by a non-invasive skin genomics platform, reported that the Journal of Drugs and Dermatology has published the results of a large registry study confirming the clinical utility of the DermTech Pigmented Lesion Assay (the "DermTech PLA") (Press release, DermTech International, MAR 5, 2020, View Source [SID1234555238]). The study also confirmed the value of DermTech’s adhesive patch by demonstrating that community-based clinicians using the DermTech PLA were able to reduce unnecessary biopsies by up to 90%, lower healthcare costs and rule out melanoma via a genomics approach that elevates pigmented lesion management beyond what the eye can see.

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"The DermTech PLA helps clinicians employ precision dermatology and harness genomics technology to detect malignant changes that are otherwise unable to be identified visually or with histopathology, reducing the probability of missing melanoma to <1%," said Ronald Moy, M.D., member of the DermTech Scientific Advisory Board and former president of the American Academy of Dermatology. "DermTech’s PLA test has the potential to elevate patient care by bringing precision genomics detection to a field that has been relying on highly subjective measures to inform biopsy decisions."

This large, yearlong utility study, conducted in the United States, included 90 providers across 53 dermatology offices and assessed 3,418 pigmented lesions clinically suspicious for melanoma with the DermTech PLA. The results of the study demonstrate that:

Clinicians use the DermTech PLA test results to guide how they practice medicine (97.53% of PLA positive lesions are surgically biopsied and 99.94% of PLA negative lesions are followed clinically and not biopsied).
90.52% (n = 3, 094) of DermTech PLA registry study test results were negative for melanoma and 9.49% (n = 324) were positive.
There is no difference in how board certified dermatologists and other licensed clinicians follow DermTech PLA results to guide biopsy decisions—potentially expanding access to care for underserved populations who often delay treatment for a disease in which early detection is critical.
The DermTech PLA identifies the gene expression associated with early melanoma by catching malignant changes a pathologist cannot see and reduces the risk of missing melanoma from 17% to less than 1%.
"Having used the DermTech PLA more than 600 times, I have seen it bring relief to people who were facing hundreds of biopsies, people concerned with facial scars and people who have experienced painful wound healing," said Brook Brouha, M.D., Ph.D., lead author on the study. "Using the PLA as a pain-free option allows providers to detect cancer before it spreads."

Up to 90% of all biopsies for suspected skin cancer are performed unnecessarily because the current standard to assess pigmented lesions remains ambiguous. These avoidable surgeries often result in multiple scars and research has shown that about 27% of older people biopsied experience poor wound healing, bleeding, or infection.

The study, "Real-world Utility of a Non-invasive Gene Expression Test to Rule out Primary Cutaneous Melanoma – A Large US Registry," is the 15th peer-reviewed publication validating the benefits of the DermTech PLA.

"This is our largest utility study to-date and these data further support the high utility of DermTech’s PLA and the high negative predictive value of this precision dermatology technology," said John Dobak, MD, Chief Executive Officer of DermTech. "The DermTech PLA can detect malignant changes in gene expression at earlier stages which can help avoid unnecessary biopsies and reduce costs."

DermTech’s PLA, which is covered by Medicare, uses RT-PCR (reverse transcriptase–polymerase chain reaction) to measure the gene expression of two genes, LINC00518 (long-intergenic non-coding RNA 00518) and PRAME (preferentially expressed antigen in melanoma) to identify the malignant changes of melanoma on the genomic level. A DermTech PLA test is positive if LINC, PRAME, or both target genes are detected as these molecular pathology findings are known to correspond with histopathology findings of in situ or invasive primary melanoma in 7%, 50% and 93% of cases, respectively.

On March 5, 2020 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, reported that it will report its fourth quarter and full year 2019 financial and operating results on Thursday, March 12, 2020, following the close of the U.S. financial markets (Press release, Lineage Cell Therapeutics, MAR 5, 2020, View Source [SID1234555237]). Lineage management will also host a conference call and webcast on Thursday, March 12, 2020, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its fourth quarter and full year 2019 financial and operating results and to provide a business update.

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Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through March 20, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 3827019.

On March 5, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported financial results for the quarter ended December 31, 2019, and provided an update on recent accomplishments and upcoming events (Press release, Syros Pharmaceuticals, MAR 5, 2020, View Source [SID1234555236]).

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"We are entering 2020 in a position of strength," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "Following the recent initiation of our Phase 1 trial of SY-5609, we are advancing two oral investigational medicines through development in multiple cancer patient populations that we believe are most likely to respond to these targeted agents, both with meaningful data readouts expected in the fourth quarter. Meanwhile, our gene control platform continues to fuel a robust preclinical and discovery pipeline in oncology and monogenic diseases. We are committed to executing with excellence on our ongoing efforts and, longer term, to leveraging our deep understanding of the regulatory genome to deliver much-needed medicines that provide profound benefits for patients."

Upcoming Milestones:

SY-1425

Report potential proof-of-concept data in the fourth quarter of 2020 from ongoing Phase 2 trial cohort evaluating SY-1425 in combination with azacitidine in RARA-positive relapsed or refractory acute myeloid leukemia (AML) patients.
Report mature data in the fourth quarter of 2020 from fully enrolled Phase 2 trial cohorts evaluating SY-1425 in combination with azacitidine in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy.
SY-5609

Report initial safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) data in fourth quarter of 2020 from dose-escalation portion of Phase 1 trial evaluating SY-5609 in patients with breast, colorectal, lung and ovarian cancers, as well as in patients with solid tumors of any histology that harbor Rb pathway alterations.
Report additional dose-escalation data, including clinical activity data, in mid-2021.
Preclinical Pipeline

Nominate next development candidate by the end of 2021.
Recent Pipeline Highlights:

In January 2020, Syros presented preclinical data showing that inhibiting cyclin-dependent kinase (CDK) 7 and CDK12 result in different transcriptional effects at the 2020 Keystone Symposia Cancer Epigenetics: New Mechanisms and Therapeutic Opportunities. The data showed that CDK12 inhibition preferentially decreases expression of genes with longer transcripts, a phenomenon not observed with CDK7 inhibition, pointing to distinct therapeutic opportunities for these two novel approaches to benefit patients with difficult-to-treat cancers. Also at Keystone, Syros presented on two new methods for identifying genes and transcriptional targets upon which specific cancers are particularly dependent for their survival.
In January 2020, Syros dosed the first patient in its Phase 1 clinical trial of SY-5609. The multi-center, open-label, dose-escalation trial is expected to enroll approximately 60 patients with breast, colorectal, lung or ovarian cancer, or with solid tumors of any histology that harbor Rb pathway alterations. The primary objectives of the trial are to assess the safety and tolerability of escalating doses of SY-5609, with the goal of establishing a maximum tolerated dose. Additional objectives include assessments of anti-tumor activity, PK, PD and predictive biomarkers of response. In a future expansion portion of the trial, multiple cohorts are planned to further evaluate the safety and anti-tumor activity of SY-5609 as both a single agent and in combination with other therapies.
In December 2019, Syros presented preclinical data demonstrating its discovery and validation of a novel fetal hemoglobin repressor, Nuclear Factor I X (NFIX) at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Data showed that, when knocked down in primary cells and an erythroid cell line expressing adult hemoglobin, NFIX induced fetal hemoglobin in nearly 100% of cells and increased total fetal hemoglobin levels to 40%, exceeding levels that are associated with a functional cure in a subset of sickle cell disease (SCD) patients.
In November 2019, Syros and its collaborators from the Whitehead Institute for Biomedical Research presented on the identification of core drivers of metastasis in triple-negative breast cancer at the 2019 San Antonio Breast Cancer Symposium.
Recent Corporate Highlights:

In February 2020, Syros announced the closing of a $60 million senior secured loan facility with Oxford Finance, LLC. Under the terms of the financing, $20 million was drawn down at closing, and $40 million will be available across two tranches, subject to certain conditions and achievement of milestones.
In December 2019, Syros appointed Mark Alles, the former Chairman and Chief Executive Officer of Celgene Corporation, to its Board of Directors. Mr. Alles is a recognized biopharmaceutical executive with a proven record of building successful global oncology organizations and commercializing innovative therapies.
In December 2019, Syros entered into a collaboration with Global Blood Therapeutics (GBT) to discover, develop and commercialize novel oral medicines for SCD and beta thalassemia. Under the agreement, Syros will use its gene control platform to identify therapeutic targets and discover drugs that induce fetal hemoglobin, and GBT will receive an option to obtain an exclusive worldwide license to develop, manufacture and commercialize products resulting from the collaboration. Under the terms of the agreement, Syros received a $20 million upfront payment. GBT is also obligated to fund up to approximately $40 million in research expenses for at least three years. Should GBT exercise its option under the agreement, Syros could receive up to $315 million in option exercise, development, regulatory, commercialization and sales-based milestones per product candidate and product resulting from the collaboration, in addition to mid- to high-single digit royalties on sales of products resulting from the collaboration.
Fourth Quarter 2019 Financial Results:

Cash, cash equivalents and marketable securities as of December 31, 2019 were $91.4 million, compared with $99.7 million on December 31, 2018. This reflects aggregate net proceeds of approximately $65 million from Syros’ two concurrent underwritten public offerings, which closed in April 2019 but does not include the $20 million upfront payment received in connection with entry into collaboration with GBT or the $20 million from the initial tranche of the Oxford loan facility.

For the fourth quarter of 2019, Syros reported a net loss of $19.7 million, or $0.46 per share, compared to a net loss of $18.0 million, or $0.54 per share, for the same period in 2018.

Revenues were $0.5 million for the fourth quarter of 2019, as compared to $0.9 million for the fourth quarter of 2018. Revenues in both the fourth quarter of 2019 and the fourth quarter of 2018 were earned under Syros’ collaboration with Incyte Corporation.
Research and development (R&D) expenses were $14.3 million for the fourth quarter of 2019, as compared to $15.1 million for the same period in 2018. This decrease was primarily attributable to the portfolio prioritization decision made during the quarter to discontinue the SY-1365 program.
General and administrative (G&A) expenses were $6.4 million for the fourth quarter of 2019, as compared to $4.4 million for the same period in 2018. This increase was primarily attributable to an increase in employee related expenses due to increased headcount, as well as stock-based compensation expense related to the accelerated vesting of certain performance-based stock options following the execution of the Global Blood Therapeutics collaboration in December 2019.
Full Year 2019 Financial Results:

For the full year ended December 31, 2019, Syros reported a net loss of $75.4 million, or $1.88 per share, compared to a net loss of $62.3 million, or $1.91 per share, for the same period in 2018.

Revenues were $2.0 million for the year ended December 31, 2019, as compared to $2.1 million for the same period in 2018. Revenues in both 2019 and 2018 related entirely to the Incyte collaboration.
R&D expenses were $58.2 million for the year ended December 31, 2019, as compared to $50.2 million for the same period in 2018. This increase was primarily attributable to the continued advancement of the Company’s existing clinical trials and advancement of its preclinical programs, including completing SY-5609 IND-enabling studies.
G&A expenses were $21.5 million for the year ended December 31, 2019, as compared to $16.2 million for the same period in 2018. This increase was primarily attributable to an increase in employee related expenses due to increased headcount.
Financial Guidance:

Based on its current plans, Syros believes that its existing cash, cash equivalents and marketable securities, including the upfront payment received in connection with the GBT collaboration and the initial tranche of the Oxford loan facility, will be sufficient to fund its planned operating expenses and capital expenditures requirements into 2022, beyond key milestones expected for both SY-1425 and SY-5609.

Conference Call and Webcast:

Syros will host a conference call today at 8:30 a.m. ET to discuss these fourth quarter and full year 2019 financial results and provide a corporate update.

To access the live conference call, please dial (866) 595-4538 (domestic) or (636) 812-6496 (international) and refer to conference ID 2764269. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the call.

On March 5, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported that the United States Patent and Trademark Office (USPTO) issued the Company its first U.S. patent related to the DecisionDx-Melanoma gene expression profile test for patients with cutaneous melanoma (Press release, Castle Biosciences, MAR 5, 2020, View Source [SID1234555235]).

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Patent No. 10,577,660, issued on March 3, 2020, covers methods of treating cutaneous melanoma in patients having high-risk cutaneous melanoma tumors. The inventors discovered that patients having high-risk cutaneous melanoma tumors can be identified by the gene-expression profile signature of their cutaneous melanoma tumors. The term of the patent extends until September 2034. This brings the total number of issued or allowed patents related to the Company’s DecisionDx-Melanoma test to 10.

"We are pleased to have been issued our first U.S. patent covering DecisionDx-Melanoma," said Derek Maetzold, Castle’s president and chief executive officer. "In the United States, we estimate approximately 130,000 patients are diagnosed with Stage I-III cutaneous melanoma each year. The clinical use of DecisionDx-Melanoma is designed to optimize post-diagnostic treatment decisions, including sentinel lymph node biopsy, frequency of follow-up and the need for imaging, which we believe will lead to improved health outcomes for patients with skin cancer."

Castle Biosciences’ DecisionDx-Melanoma test is a 31-gene expression profile prognostic test for cutaneous melanoma that predicts 5-year risk of metastasis as low risk (Class 1, 1A lowest risk) or high risk (Class 2, 2B highest risk), as well as metastasis to the sentinel lymph node, based on an individual patient’s tumor biology.

About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node positivity, independent of traditional staging factors, and has been studied in more than 5,700 patient samples. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in four archival risk of recurrence studies of 901 patients and six prospective risk of recurrence studies including more than 1600 patients. Prediction of the likelihood of sentinel lymph node positivity has also been validated in two prospective multicenter studies that included more than 3,000 patients. Impact on patient management plans for one of every two patients tested has been demonstrated in four multicenter and single-center studies including more than 560 patients. The consistent performance and accuracy demonstrated in these studies provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. Since its commercial launch through December 31, 2019, DecisionDx-Melanoma has been ordered 51,967 times for use in patients with cutaneous melanoma.

More information about the test and disease can be found at www.SkinMelanoma.com.

On March 5, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported financial results for the year ended December 31, 2019, and provides an update on the Company’s recent pipeline developments, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAR 5, 2020, View Source [SID1234555234]).

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"2019 was a pivotal year for Rocket marked by multiple clinical and regulatory achievements across the pipeline," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We now have four gene therapy programs in the clinic, have established proof of concept for our lenti pipeline utilizing our commercial-grade ‘Process B’ manufacturing in both FA and LAD-I and have treated the first three patients in our Phase 1 trial for Danon Disease."

Dr. Shah continued, "We are excited to build upon the momentum by bringing our fifth program to the clinic, advancing FA and LAD-I with BLA/MAA filings commencing in the next two to three years and completing our R&D and manufacturing facility. In addition to reporting progress from our FA and LAD-I programs, we’re looking forward to presenting initial proof of concept data from PKD and Danon this year. With these anticipated milestones by year end, we are even closer to making our gene therapies available to patients and families with rare disease."

Full Year 2019 and Recent Pipeline Developments

Rocket expands footprint in Cranbury, New Jersey. Rocket’s Research & Development (R&D) and Chemistry, Manufacturing and Controls (CMC) operations will be housed in a new 103,720 square foot facility, of which 50,000 square feet will be dedicated to adeno-associated virus (AAV) Current Good Manufacturing Practice (cGMP) manufacturing. The manufacturing facility will result in a one-time additional spend of $30 million in the first half of 2020. With construction underway, Rocket has secured adequate supply of cGMP AAV9 to commercialization in partnership with a contract manufacturing organization (CMO) and established an agreed path forward with the Agency using the current process. Occupancy is anticipated in the first half of 2020, with first cGMP clinical product release expected in 2021.
Four gene therapy programs have entered the clinic. With the initiation of the global Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD) in the fourth quarter, Rocket has advanced four gene therapy candidates into the clinic. In December, the Company announced treatment of the first patient in the global registrational Phase 2 study of RP-L102 "Process B" for Fanconi Anemia (FA), representing the launch of the Company’s first Phase 2 trial. A Phase 1/2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I) and a Phase 1 trial of RP-A501 for Danon Disease are ongoing. Data updates from each of these programs as well as for PKD are expected throughout 2020.
Proof of concept for the lenti pipeline established using "Process B". "Process B" is an optimized manufacturing method which incorporates a modified stem cell enrichment process, transduction enhancers, as well as commercial-grade vector and final drug product. Preliminary Phase 1 data highlight the potential of RP-L102 "Process B" in treating FA. "Process B" allows for consistent drug product across patients and a vector copy number (VCN) two to three-fold higher than that administered to optimally-treated "Process A" patients. These patients also demonstrated early signs of engraftment and bone marrow restoration. Results from the Phase 1/2 trial of LAD-I also establish the potential of this manufacturing process, with preliminary data from the first patient treated showing drug product VCN of 3.8, a 3-month post-treatment myeloid VCN of 1.5 and a 3-month post-treatment CD18 expression level of 45%, a clear increase as compared to the pre-treatment level of < 1%.
Global registrational Phase 2 trial of RP-L102 for FA is underway. The first patient has been treated in the global registrational Phase 2 trial of RP-L102 for FA and enrollment continues. The study initiation follows alignment from the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) on trial design and the primary endpoint of resistance to mitomycin-C (MMC), a measure of bone marrow cell correction. The global trial will enroll 10 patients from the U.S. and EU. Patients will receive a single intravenous infusion of RP-L102 that utilizes fresh cells and "Process B". The primary endpoint of improved MMC-resistance may also serve as a surrogate endpoint for accelerated approval. Preliminary data are expected in the second half of 2020.
Enrollment continues in Phase 1 trial of RP-L301 for PKD. The global, open-label, single-arm clinical trial will enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. The trial is designed to assess the safety, tolerability and preliminary efficacy of RP-L301. Preliminary data are anticipated in the second half of 2020.
Three patients have been treated in the low dose cohort in the Phase 1 clinical trial of RP-A501 for Danon Disease. No major safety concerns have been observed in any of the three patients treated. Phase 1 data are expected in the second half of 2020.
Three regulatory designations were awarded across the pipeline, including PRIME, Fast Track and Rare Pediatric Disease. RP-L102 for FA received EMA PRIority MEdicines (PRIME) eligibility, RP-L301 for PKD received Fast Track designation from the FDA and RP-L401 for Infantile Malignant Osteopetrosis (IMO) received Rare Pediatric Disease designation from the FDA. Each designation provides numerous incentives to support the development of Rocket’s programs, including increased access to regulatory authorities and expedited development and review timelines.
Company expands research and development collaborations for LAD-I. Rocket announced a partnership with the University of California, Los Angeles (UCLA) to lead U.S. clinical development efforts for LAD-I. The Company also received a $6.5 million grant from the California Institute for Regenerative Medicine (CIRM) to support the Phase 1/2 registrational clinical trial of RP-L201 for LAD-I.
Rocket strengthens its balance sheet by extending the maturity of the existing convertible notes and securing ~$188 million in two public equity offerings. Rocket extended the maturity of the 5.75% convertible notes due August 2021. The Company conducted an exchange offering and successfully exchanged $39.35 million of the outstanding $52 million convertible notes. The new notes will mature on August 1, 2022 and have an interest payment of 6.25% per annum. The remainder of the notes will retain the existing maturity date and interest payment. Additionally, in 2019 the Company closed two oversubscribed underwritten public offerings of common stock for gross proceeds of approximately $188 million.
Anticipated Milestones

FA (RP-L102)
Additional data update (2Q)
Preliminary Phase 2 data (2H)
Danon Disease (RP-A501)
Danon Disease day (1Q)
Cohort 1 complete (1Q)
Advancing to next cohort (2Q)
Phase 1 data (2H)
LAD-I (RP-L201)
Phase 1 data update (2H)
Initiate Phase 2 study (2H)
PKD (RP-L301)
First patient treatment (2Q)
Preliminary Phase 1 data (2H)
IMO (RP-L401)
Initiation of clinical study (2H)
Upcoming Investor Conferences

Annual Barclays Global Healthcare Conference—March 11, 2020 in Miami, F.L.
Oppenheimer’s 30th Annual Healthcare Conference—March 18, 2020 in New York, N.Y.
2nd Annual Guggenheim Genomic Medicines and Rare Disease Day—April 3, 2020 in New York, N.Y.
Fourth Quarter and Full Year 2019 Financial Results

Cash position. Cash, cash equivalents and investments as of December 31, 2019, were $304.1 million.
Debt. Our balance sheet includes $52.0 million of fully convertible notes.
R&D expenses. Research and development expenses were $14.7 million and $58.6 million for the three and twelve months ended December 31, 2019, compared to $23.7 million and $53.3 million for the three and twelve months ended December 31, 2018. The increase in research and development expenses for the twelve months ended December 31, 2019, was primarily driven by an increase in clinical trial expenses and an increase in compensation expense due to increased headcount. The decrease in research and development expenses for the three months ended December 31, 2019 was primarily due to a one time license fee payment made in the three months ended December 31, 2018.
G&A expenses. General and administrative expenses were $5.0 million and $17.5 million for the three and twelve months ended December 31, 2019, compared to $2.9 million and $17.9 million for the three and twelve months ended December 31, 2018. The increase in general and administrative expenses for three months ended December 31, 2019, was primarily driven by an increase in compensation expense and non-cash stock-based compensation expense due to increased headcount.
Net loss. Net loss was $19.9 million and $77.3 million or $0.39 and $1.58 per share (basic and diluted) for the three and twelve months ended December 31, 2019, compared to $27.3 million and $74.5 million or $0.66 and $1.89 per share (basic and diluted) for the three and twelve months ended December 31, 2018.
Shares outstanding. 54,773,061 shares of common stock were outstanding as of December 31, 2019.
Financial Guidance

Cash position. As of December 31, 2019, we had cash, cash equivalents and investments of $304.1 million. Rocket expects such resources will be sufficient to fund its operations into 2022.