On May 14, 2020 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported that data from the Phase 1 study of SRF231, a fully human, high-affinity anti-CD47 antibody, will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Annual Meeting, to be held virtually May 29-31 (Press release, Surface Oncology, MAY 14, 2020, View Source [SID1234558047]).

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A summary is provided below; the full poster will be placed on Surface Oncology’s website following the presentation.

Presentation Type: e-poster (Abstract: 3064 / Poster: 128)
Title: Results of a first-in-human Phase 1 study of SRF231, a fully human, high-affinity anti-CD47 antibody
Lead Author: Amita Patnaik
Session: Developmental Therapeutics – Immunotherapy

In 2018, Surface Oncology deprioritized the SRF231 clinical program and is concluding its Phase 1 study.

On May 14, 2020 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported financial results for the first quarter of 2020 and provided a business update (Press release, Sellas Life Sciences, MAY 14, 2020, View Source [SID1234558046]).

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"The first quarter of 2020 brought unprecedented times with the onset of the COVID-19 global pandemic. Our key priorities are to focus on the health and safety of our patients, investigators, employees and other stakeholders as well as diligently executing our business operations. We have been fortunate to be able to continue to progress our clinical development program for galinpepimut-S (GPS), including our Phase 3 REGAL study in acute myeloid leukemia (AML), our Phase 1/2 basket study in combination with pembrolizumab (KEYTRUDA), and our Phase 1 trial in combination with nivolumab (Opdivo) in malignant pleural mesothelioma (MPM) despite the uncertainty and disruption caused by the pandemic. Our activities around the REGAL study, including those for additional site activations in the United States and Europe, have continued unabated. Screening is continuing in the basket study and dosing of enrolled patients is continuing in the MPM Phase 1 study," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Additionally, we are continuing our business development efforts to pursue out-licensing opportunities for further clinical development of nelipepimut-S (NPS)."

First Quarter 2020 and Recent Pipeline Highlights

Galinpepimut-S (GPS) Program

In April 2020, SELLAS announced the formation of the Steering Committee for its Phase 3 REGAL clinical trial of GPS in patients with AML who have achieved complete remission after second-line anti-leukemic therapy (CR2). The Steering Committee currently consists of three members: Dr. Hagop Kantarjian, MD, Professor and Chair of the Department of Leukemia at The University of Texas MD Anderson Cancer Center, and Principal Investigator at MD Anderson for the multi-center Phase 3 REGAL study and Chair of the REGAL Steering Committee; Dr. Javier Pinilla-Ibarz, MD, PhD, Director of Immunotherapy for Malignant Hematology at the H. Lee Moffitt Cancer Center and member of the SELLAS Scientific Advisory Board; and Dr. Moshe Yair Levy, MD, Director of Hematologic Malignancies at the Texas Oncology – Baylor Charles A. Sammons Cancer Center.
In February 2020, SELLAS announced positive follow-up data from its Phase 1/2 study of GPS in CR2 AML patients. The final data show a median overall survival (OS) of 21.0 months, at a median follow-up of 30.8 months, in patients receiving GPS compared to 5.4 months in patients treated with best standard care (p-value < 0.02). GPS therapy continued to be well-tolerated throughout the study.
In February 2020, SELLAS announced the enrollment of the first patient in an investigator-sponsored clinical trial of GPS in combination with Bristol-Myers Squibb’s anti-PD-1 therapy, nivolumab (Opdivo), in patients with MPM.
In January 2020, SELLAS announced that it commenced patient screening for its pivotal Phase 3 REGAL study of GPS in patients with AML in CR2.
Nelipepimut-S (NPS) Program

In March 2020, SELLAS announced preliminary antigen-specific immune response data from a Phase 2 randomized investigator-sponsored trial of NPS in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) in women with ductal carcinoma in situ (DCIS) of the breast who are HLA-A2+ or A3+ positive, express HER2 at IHC 1+, 2+, or 3+ levels, and are pre- or post-menopausal. Preliminary data show an 11-fold increase in a CD8 cytotoxic T-lymphocytes immune response in patients who received a single dose of NPS compared to baseline. The final data is being further analyzed by the National Institute of Health, MD Anderson Cancer Center and the study principal investigator, Dr. Elizabeth Mittendorf, MD, PhD of the Dana-Farber/Brigham and Women’s Cancer Center, and will be presented at an upcoming medical conference.
In March 2020, SELLAS announced that final results from the efficacy and safety data analysis of the Phase 2b independent investigator-sponsored clinical trial of the combination of trastuzumab (Herceptin) +/- NPS targeting HER2 low-expressing breast cancer patient cohorts, including patients with triple negative breast cancer (TNBC), were recently published in the peer reviewed journal, Clinical Cancer Research. With regard to the TNBC patient cohort, the data analysis shows:
– Disease-free survival (DFS) landmark rate at 24 months for patients treated with NPS plus trastuzumab (n=53) was 92.6% compared to 70.2% for those treated with trastuzumab alone (n=44), a clinically and statistically significant improvement.
– There was a statistically significant reduction of 71.9% (p=0.01) in the frequency of clinically detected recurrences in patients treated with the combination (NPS plus trastuzumab) versus trastuzumab alone.
– The combination was generally well-tolerated and there were no clinicopathologic differences between the study groups.
In February 2020, SELLAS announced that it had finalized the design and plan for a Phase 3 registration-enabling study of NPS in combination with trastuzumab for the treatment of patients with TNBC in the adjuvant setting after standard treatment, following feedback from a Type C review with the FDA. SELLAS is actively pursuing out-licensing opportunities to fund and conduct the future clinical development of NPS.
Recent Corporate Highlights

In April 2020, SELLAS retained PCG Advisory Inc., a leading investor relations and digital strategies firm, to serve as an advisor for investor relations and strategic communications.
In February 2020, SELLAS announced the appointment of Dragan Cicic, MD, as Senior Vice President, Clinical Development.
First Quarter 2020 Financial Results

Cash Position: As of March 31, 2020, cash and cash equivalents totaled approximately $6.7 million, compared to $2.6 million as of March 31, 2019. Net cash used in operating activities was $6.8 million for the three months ended March 31, 2020, compared to $5.0 million for the three months ended March 31, 2019. Net cash provided by financing activities was $6.3 million for the three months ended March 31, 2020, which was attributable to $6.0 million in net proceeds from a registered direct offering of shares of common stock and warrants to purchase common shares in a concurrent private placement consummated in January 2020, and $0.3 million from the collection of a stock subscription receivable. During the three months ended March 31, 2019, the Company generated $2.2 million of net cash from financing activities from the exercise of certain common stock warrants.

R&D Expenses: Research and development expenses were $1.9 million for the first quarter of 2020, as compared to $1.9 million for the three months ended March 31, 2019. While relatively unchanged from the previous year quarter, for the three months ended March 31, 2020 there was a $0.2 million increase in clinical trial expenses, a $0.1 million increase in manufacturing related expenses for GPS due to the Company’s ongoing basket trial of GPS in combination with pembrolizumab and the Phase 3 REGAL study of GPS as a monotherapy in AML, and a $0.1 million increase in other research and development expenses, partially offset by a $0.2 million decrease in personnel related expenses due to reduced headcount and a $0.2 million decrease in licensing fees.

G&A Expenses: General and administrative expenses were $2.2 million for the three months ended March 31, 2020, as compared to $2.5 million for the three months ended March 31, 2019. The $0.3 million decrease was primarily due to a $0.3 million decrease in legal fees, a $0.2 million decrease in accounting fees, and a $0.1 million decrease in personnel related expenses due to reduced headcount. These decreases were partially offset by a $0.2 million increase in premiums for directors and officers liability insurance and a $0.1 million increase in outsourced consulting and public company costs.

Net Loss: Net loss attributable to common stockholders was $4.2 million for the first quarter of 2020, or a basic and diluted loss per share attributable to common stockholders of $0.66, as compared to a net loss attributable to common stockholders of $5.0 million for the first quarter of 2019, or a basic and diluted loss per share attributable to common stockholders of $11.12.

On May 14, 2020 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biotechnology company targeting cancers caused by dysregulated gene expression, reported its corporate and financial results for the first quarter ended March 31, 2020 (Press release, Salarius Pharmaceuticals, MAY 14, 2020, View Source [SID1234558045]).

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Financial Highlights:

•Closed $11 million gross proceeds in an underwritten public offering
•Three-month period ended March 31, 2020 net loss per common share – basic and diluted – for continuing operations of $0.22, compared to $0.64 for the same period ended March 31, 2019
•Total cash and cash equivalents of $9.65 million as of March 31, 2020
•Up to $9.10 million remains available to draw from the Cancer Prevention and Research Institute of Texas (CPRIT) Award, upon meeting certain requirements
•National Pediatric Cancer Foundation (NPCF) continues to provide significant funding for Ewing sarcoma study

Recent Business and Corporate Highlights:

•Phase 1/2 clinical study of seclidemstat in Ewing sarcoma advanced into the sixth level dosing cohort with Maximum Tolerated Dose (MTD) expected to be reached in mid-2020
•Scientific paper published highlighting potential of combining seclidemstat with cancer immunotherapies
•In vitro data shows that seclidemstat helps overcome tumor resistance to checkpoint inhibitors
•European Patent Office (EPO) issued a notice of allowance for Patent EP274430 for seclidemstat
•William "Bill" McVicar, Ph.D., named as new Chairman of the Board of Directors

"Salarius is anticipating the potential for multiple clinical data releases and value building events throughout 2020 and beyond," said David Arthur, President and CEO of Salarius. "Our clinical programs for seclidemstat continue to progress, and in the first quarter, we advanced dose-escalation in our Phase 1/2 clinical trial of seclidemstat in Ewing sarcoma. We eagerly anticipate releasing data from both the Ewing sarcoma clinical trial and our second clinical trial in advanced solid tumors (AST) in 2020."

Mr. Arthur continued, "Salarius has worked to adapt to the unexpected and challenging circumstances resulting from the COVID-19 pandemic. At this time, we are experiencing minimal COVID-19 disruptions to our ongoing clinical programs and continue to enroll patients in our clinical trials."

Mr. Arthur concluded, "Our goal is to maximize the potential of seclidemstat and bring hope to patients fighting rare, pediatric and other cancers. To that end, Salarius is well-capitalized and has the resources, including $9.1 million in non-dilutive funding that remains available from the 2016 $18.7 million CPRIT award, that we believe will advance both our Ewing sarcoma and AST programs, as well as early research on the potential for combining seclidemstat with immunotherapies, well into 2021."

Three-Month Financial Results:
For the three-month period ended March 31, 2020, Salarius’ reported net loss was $2.08 million, or $0.22 per basic and diluted share, compared to a net loss of $1.52 million, or $0.64 per basic and diluted share for the same period in 2019. The loss before other income for the three-month period ended March 31, 2020 increased by $0.84 million compared to the loss before other income for the same time span last year, which was primarily due to an increase of $0.94 million in research and development expenses resulting from increased clinical trial expenses and drug manufacturing costs. Salarius also reported a net increase of $0.37 million in general and administrative costs resulting from Salarius’ transformation into a public company and increased personnel expenses during the current quarter, somewhat offset by lower professional fees and legal costs compared to same period in 2019.

As of March 31, 2020, total cash, cash equivalents and restricted cash was $9.65 million, compared to $5.77 million as of March 31, 2019 and $3.74 million at year-end 2019.

$11 Million Underwritten Public Offering
On February 11, 2020, Salarius completed a public offering with total gross proceeds of approximately $11 million, prior to deducting underwriting discounts and commissions and offering expenses payable by Salarius. Salarius intends to use the net proceeds from the offering for general working capital purposes.

Summary of Corporate and Operational Events:

Ewing Sarcoma Dose Escalation Clinical Trial Advances into Sixth Dosing Cohort
Salarius is conducting two Phase 1/2 clinical trials for the company’s lead investigational drug candidate, seclidemstat – one in patients with relapsed or refractory Ewing sarcoma and the second in patients with advanced solid tumors (AST) resistant to standard-of-care therapies. The trials are designed as open-label dose-finding studies with a primary objective to characterize the pharmacokinetics (PK), Maximum Tolerated Dose (MTD), and initial safety profile of seclidemstat and a secondary objective to assess the preliminary efficacy.

In Q1 2020, the Safety Review Committee overseeing the Ewing sarcoma clinical trial approved the advancement of the study to the sixth dosing cohort (1,200mg BID) out of seven potential cohorts, and patient enrollment is ongoing. Thus far, PK data from the trial suggest that plasma drug levels measuring the concentration of seclidemstat in a patient’s plasma remain dose proportional and there is no evidence of a plateau in exposure levels.

Dose escalation continues with the Phase 1/2 clinical trial in AST, which is now in the fourth dosing cohort (600mg BID) out of seven possible cohorts. The AST study enrolls patients with a focus on prostate, breast, ovarian, melanoma, colorectal, non-Ewing sarcomas and other cancers where seclidemstat demonstrated single-agent preclinical activity.

Based on current projections, Salarius believes both clinical trials are on track to reach MTD in 2020, and shortly after, begin the dose-expansion phase of each study. Salarius expects to report early safety and pharmacokinetic data before year-end 2020.

Scientific Paper Highlights Use of Seclidemstat in Combination with Checkpoint Inhibitors
Salarius is exploring additional indications for seclidemstat to expand the drug’s market potential, and this includes seclidemstat’s potential for use in combination with a type of cancer immunotherapy commonly known as checkpoint inhibitors. Checkpoint inhibitors, estimated at $15 billion in annual global sales, are designed to unleash an immune attack on cancer cells. However, these therapies do not work in about 70% of cancer patients, and in patients who do show an initial response, many suffer a return of the disease.

On February 4, 2020, a scientific paper entitled, "The Novel Reversible LSD1 Inhibitor SP-2577 Promotes Anti-Tumor Immunity in SWItch/Sucrose-NonFermentable (SWI/SNF)1," was published on bioRxiv.com. The paper highlighted data from in vitro studies conducted by Sunil Sharma, M.D., Salarius’ scientific founder, and his team at the Translational Genomic Institute (TGen) in Phoenix, Ariz., that demonstrate the potential for seclidemstat (also known as SP-2577) to be used in combination with checkpoint inhibitors to treat cancers with identifiable mutations in proteins of the SWI/SNF complex. In this study, seclidemstat modulated the tumor microenvironment to help overcome the resistance to checkpoint inhibitors

The SWI/SNF complex plays an important role in modulating gene expression, and mutations in proteins of the SWI/SNF complex occur in roughly 20% of human cancers. In this study, Dr. Sharma’s team investigated the ability of seclidemstat to promote anti-tumor immunity and T-cell infiltration in two types of ovarian cancer – small cell carcinoma of the ovary hypercalcemic type and ovarian clear cell carcinoma — that both carry mutations in proteins of the SWI/SNF complex.

Salarius Strengthens IP Portfolio Around Seclidemstat
On March 4, 2020, Salarius announced the continued enhancement of the U.S. and global intellectual property (IP) portfolio governing seclidemstat. The European Patent Office issued a notice of allowance for Patent EP274430 exclusively licensed to Salarius from the University of Utah Research Foundation indicating that the agency is satisfied that the patent application meets all EPO requirements.

In all, Salarius holds 22 issued patents in the U.S. and abroad. The company’s current IP estate includes five patents issued in the U.S. and another 17 patents issued in Europe, Australia, Brazil, China, Eurasia, Israel, India, Japan, Korea, Mexico, New Zealand, Singapore, and South Africa. Meanwhile, Salarius has 11 patent applications pending approval in Europe, Brazil, Canada, Israel, India, Korea, Mexico, Singapore, and China. All 22 issued patents and the 11 pending applications are directed to seclidemstat or structurally similar compounds.

Salarius Names New Chairman of the Board
On April 29, 2020, Salarius announced that Board member William "Bill" McVicar, Ph.D., was named by the Board as its new Chairman. Dr. McVicar is a seasoned pharmaceutical industry executive with more than 30 years of clinical development experience. He recently served as Chief Executive Officer of Flex Pharma, and previously served as Chief Scientific Officer and President at Inotek Pharmaceuticals.

Conference Call Information:
Salarius Pharmaceuticals will host a conference call and live audio webcast on Thursday, May 14, 2020, at 4:30 p.m. ET, to discuss its corporate and financial results for the first quarter 2020. Interested participants and investors may access the conference call by dialing either:

An audio webcast will be accessible via the Investors Events and Presentations section of the Company’s website View Source An archive of the webcast will remain available for 90 days beginning at approximately 6:00 p.m. ET, on May 14, 2020.

On May 14, 2020 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company focused on developing targeted therapies to inhibit frontier cancer targets, reported its financial results for the first quarter ended March 31, 2020, and provided an update on its R&D pipeline and other corporate developments (Press release, Revolution Medicines, MAY 14, 2020, View Source [SID1234558044]).

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Highlights from the Quarter Ended March 31, 2020

"Revolution Medicines achieved important scientific, clinical and operational milestones during this quarter," said Mark A. Goldsmith, M.D., Ph.D., president, chief executive officer and chairman of Revolution Medicines. "In January, we presented initial data from our ongoing Phase 1 monotherapy trial evaluating our investigational new drug designed to inhibit SHP2, RMC-4630, in patients with KRAS mutant non-small cell lung cancer (NSCLC) at the AACR (Free AACR Whitepaper)-IASLC International Joint Conference. The findings represent the first reported evidence of clinical activity against KRAS mutant lung cancers by a SHP2 inhibitor, as well as initial evidence of the potential benefit of an intermittent dosing schedule. Our ongoing Phase 1/2 clinical program evaluating RMC-4630 in a range of tumor types continues to advance and enrollment has been in line with our expectations. We also continued to make progress across our broad preclinical pipeline that supports our strategy to target multiple nodes in the oncogenic RAS pathway and bring forward potential monotherapies and combination treatment regimens. During the period, the company also completed a successful IPO, raising gross proceeds of more than $273 million. Revolution Medicines’ strong balance sheet will support continued development of our promising pipeline on behalf of cancer patients."

"We acknowledge the severe health and economic impact of the COVID-19 pandemic we are all experiencing and the burden it has placed on our healthcare system and the clinical trial landscape. Early on, Revolution Medicines made appropriate adjustments to our operating approach, and we’ve continued to make progress on both our preclinical and clinical programs. At present, we do not expect material delays in our ongoing clinical trials, but it is reasonable to anticipate that for planned future studies some site initiations may be delayed, and enrollment may be slowed for some period of time. We are continuing to refine our approach as needed to minimize these impacts."

Scientific and Clinical Highlights

Revolution Medicines demonstrates first ever clinical activity against KRAS mutant lung cancers with SHP2 inhibitor – In January 2020, at the 6th AACR (Free AACR Whitepaper)-IASLC International Joint Conference, the company presented preliminary evidence demonstrating that RMC-4630, the company’s investigational SHP2 inhibitor, showed initial clinical activity in patients with NSCLC bearing KRAS mutations, particularly KRASG12C. Findings also demonstrated the potential benefit of an intermittent RMC-4630 dosing schedule.

RMC-4630 clinical program continues to advance – Revolution Medicines continues to explore optimal dosing and scheduling of RMC-4630 in both its ongoing Phase 1 monotherapy and Phase 1b/2 combination therapy trials. The company plans to expand its RMC-4630 combination therapy program and is prepared for the initiation of new studies evaluating the compound in combination with Amgen’s investigational KRASG12C(OFF) inhibitor, AMG 510, with the EGFR inhibitor osimertinib (Tagrisso), and with a PD-1 inhibitor. While the COVID-19 pandemic may indirectly cause some delays in the initiation of new clinical studies, the company currently expects enrollment in these studies to begin in 2020.

RMC-5552 – IND-enabling work continuing – RMC-5552, the company’s potent and selective inhibitor of mTORC1, continues to advance through investigational new drug (IND)-enabling development. The company remains on track to be IND-ready with this compound by the end of 2020.

Mutant-selective RAS(ON) inhibitor program advancing; development candidate to be nominated – Revolution Medicines is developing a portfolio of mutant-selective RAS(ON) inhibitors that it believes may be the first potent, selective, cell-active inhibitors of the active, GTP-bound form of RAS, or RAS(ON). The company continues to make significant progress toward optimizing key properties of such inhibitors. In line with previous projections, the company continues to anticipate nomination of its first development candidate from this portfolio in 2020.

SOS1 inhibitor program advances into lead optimization. Revolution Medicines’ program targeting SOS1, a protein that plays a central role in driving oncogenic signals through the RAS pathway, continues to advance. Our growing collection of potent and selective inhibitors exhibiting attractive preclinical profiles has enabled the program to progress into the lead optimization stage in pursuit of a development candidate.

Findings recently published in Cancer Research support combination of RMC-4630 with an anti-PD-1 antibody – In a paper published on April 29, 2020, Revolution Medicines researchers described ways in which a SHP2 inhibitor enhances the immune response to tumors, representing a second group of anti-tumor mechanisms beyond its direct effects within cancer cells themselves. The paper also reported deep and durable tumor growth inhibition following combination treatment with a SHP2 inhibitor and anti-PD-1 inhibitor in mouse cancer models, yielding complete tumor regressions and sustained immunological memory.

Multiple abstracts selected for presentation at upcoming American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II – Revolution Medicines has been notified that four of the company’s submissions have been selected for presentation at the upcoming virtual AACR (Free AACR Whitepaper) meeting scheduled to take place in June 2020. Titles and abstracts of these presentations will be disclosed by AACR (Free AACR Whitepaper) on May 15, 2020.

Anticipated Scientific and Clinical 2020 Milestones

Clinical data update from RMC-4630 program

Clinical trial initiations:

RMC-4630 combination with AMG 510

RMC-4630 combination with osimertinib

RMC-4630 combination with PD-1 inhibitor

Nomination of first development candidate for RAS(ON) inhibitor program

RMC-5552 IND-readiness

Corporate Highlights

Closed initial public offering – In February 2020, Revolution Medicines closed its initial public offering of 16,100,000 shares of common stock, including the exercise in full by the underwriters of their option to purchase an additional 2,100,000 shares of common stock, at a public offering price of $17.00 per share. The gross proceeds from the offering were $273.7 million, before deducting underwriting discounts, commissions and other offering expenses payable by Revolution Medicines.

The company expects to use the net proceeds from this offering to fund the development of its multiple RAS inhibitor programs, including the RAS(ON) portfolio, SOS1 inhibitor program, and 4EBP1/mTORC1 program, and other general corporate purposes, which may include the hiring of additional personnel, capital expenditures and the costs of operating as a public company.

USPTO grants key RMC-4630 patent – In March 2020, the United States Patent and Trademark Office issued U.S. Patent No. 10,590,090 to the company, providing, in part, composition of matter protection for its SHP2 inhibitors, including RMC-4630.

Facilities Expansion – To support Revolution Medicines’ expanding operations, the company completed a lease transaction in April 2020 that will provide an additional 19,483 square feet in Redwood City, CA. The company campus now includes two buildings that house office, laboratory and research and development space.

Q1 2020 Financial Highlights

Cash Position: Cash, cash equivalents and marketable securities were $347.9 million as of March 31, 2020, compared to $122.8 million as of December 31, 2019. The increase was primarily due to proceeds from the IPO in February 2020.

Revenue: Total revenue, consisting of revenue from our collaboration agreement with Sanofi, was $11.5 million for the quarter ended March 31, 2020, compared to $13.2 million for the quarter ended March 31, 2019. This decrease was primarily due to lower reimbursed research and development services in the quarter ended March 31, 2020 resulting from lower manufacturing costs. During the quarter ended March 31, 2019, we incurred upfront manufacturing costs related to the supply of RMC-4630 for our clinical trials.

R&D Expenses: Research and development expenses were $27.5 million for the quarter ended March 31, 2020, compared to $21.2 million for the quarter ended March 31, 2019. This increase was primarily due to an increase in research expenses associated with our RAS inhibitor programs.

G&A Expenses: General and administrative expenses were $5.2 million for the quarter ended March 31, 2020, compared to $2.4 million for the quarter ended March 31, 2019. This increase was primarily due to an increase in expenses associated with transitioning to and becoming a public company.

Net Loss: Net loss was $19.5 million for the quarter ended March 31, 2020, compared to net loss of $10.1 million for the quarter ended March 31, 2019.

On May 14, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, Regulus, MAY 14, 2020, View Source [SID1234558043]).

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"We have completed the second cohort and have initiated the dosing for the third and final cohort in the Phase 1 multiple ascending dose ("MAD") clinical study for RGLS4326" said Jay Hagan, CEO of Regulus. "Additionally, preparations for the Phase 1b study in patients with autosomal dominant polycystic kidney disease ("ADPKD") are well underway with plans to initiate in the second half of 2020."

Program Highlights

Initiated Dosing of the Third and Final Cohort in RGLS4326 Phase 1 for ADPKD: In April 2020, the Company initiated dosing of the third and final cohort of the MAD clinical study of RGLS4326, a novel oligonucleotide designed to inhibit miR-17 for the treatment of ADPKD. The Company expects to complete this study in mid-2020 with topline results available thereafter. The Company is planning to initiate a Phase 1b short-term dosing study in patients with ADPKD in the second half of 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity.

Financial Results

Cash Position: As of March 31, 2020, Regulus had $28.1 million in cash and cash equivalents.

Research and Development (R&D) Expenses: R&D expenses were $3.1 million for the three months ended March 31, 2020, compared to $6.0 million for the same period in 2019. The aggregate decrease was driven by a $1.5 million reduction in personnel and internal expenses and a $1.0 million reduction in external development expenses, both of which were primarily attributable to a reduction in costs associated with the partial clinical hold of the RGLS4326 MAD study. In December 2019, the U.S. Food and Drug Administration ("FDA") lifted the partial clinical hold on the MAD study and it was recommenced in February 2020.

General and Administrative (G&A) Expenses: G&A expenses were $2.4 million for the three months ended March 31, 2020 compared to $3.5 million for the same period in 2019. These amounts reflect personnel-related and ongoing general business operating costs. The decrease is primarily attributable to continued cost reduction efforts subsequent to our corporate restructuring in the third quarter of 2018.

Revenue: Revenue was less than $0.1 million for the three months ended March 31, 2020. Revenue was $6.8 million for the three months ended March 31, 2019, attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Net Loss: Net loss was $5.9 million, or $0.25 per share (basic and diluted), for the three months ended March 31, 2020, compared to $3.3 million, or $0.31 per share (basic and diluted), for the same period in 2019.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by the U.S. Food and Drug Administration until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.