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Caladrius Biosciences Provides Corporate Update and Reports 2020 Third Quarter Financial Results

On November 5, 2020 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse disease, reported financial results for the three and nine months ended September 30, 2020 (Press release, Caladrius Biosciences, NOV 5, 2020, View Source [SID1234570068]).

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"Amid the continuing global impact of COVID-19, our team continues to rise to the occasion, addressing a multitude of challenges tied to the pandemic," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "To date in 2020, we have extended our cash runway through the end of 2021, while continuing to deliver on a number of key initiatives in support of our clinical programs including the initiation of our study of CLBS119 for the treatment of COVID-19 induced lung damage and finalizing preparations for the start to our newly-named Phase 2b FREEDOM Study of CLBS16 in coronary microvascular dysfunction."

"We are excited about what lies ahead in 2020 and expect to build on this momentum as we continue to advance our clinical development pipeline and strive to achieve a number of important development milestones throughout the balance of the year," concluded Dr. Mazzo.

Product Development and Financing Highlights

HONEDRA (formerly CLBS12) development in Japan continues to progress

The Company’s open-label, registration-eligible study in Japan of HONEDRA (formerly CLBS12), continues to progress toward enrollment completion, although enrollment has been slowed by the impact of the COVID-19 pandemic in Japan. HONEDRA is a SAKIGAKE-designated product candidate for the treatment of critical limb ischemia ("CLI"); a disease with limited treatment options – most of which have minimal impact – and a higher combined incidence and mortality rate than all cancers but lung cancer. As previously reported, the Buerger’s Disease (an "orphan-sized" type of CLI) cohort has concluded with 4 out of 7 (~60%) patients achieving a positive outcome, an outstandingly positive result for these patients who normally see continued progression leading to amputation. The Company remains encouraged by the patient pre-screening pipeline that has been identified for the arteriosclerosis obliterans ("ASO") cohort, which is the primary arm of the study, and anticipates trial enrollment to conclude in the first quarter of 2021, leading to top line data for the full study in late 2021 or early 2022.

CLBS14 remains poised to enter a single confirmatory phase 3 clinical trial

The Company’s Phase 3 protocol for its RMAT-designated product candidate, CLBS14, for the treatment of no-option refractory angina ("NORDA") remains ready to initiate pending sufficient funding to run the program to completion. Based on an abundance of very strong data from previous Phase 1, 2, and 3 studies, Caladrius remains confident in the potential for clinical success once the program is executed.

CLBS16 to be studied in Phase 2b trial for the treatment of coronary microvascular dysfunction

Caladrius recently completed and announced the results of its ESCaPE-CMD Phase 2a study of CLBS16 for the treatment of coronary microvascular dysfunction ("CMD"), a disease that continues to be underdiagnosed and potentially afflicts millions annually – a vast majority of whom are female – with no current treatment options. Data from the Phase 2a trial showed a positive therapeutic effect with a statistically significant improvement in angina frequency, coronary flow reserve, Canadian Cardiovascular Society Angina Class and Seattle Questionnaire score, as well as an acceptable safety profile. The Company is committed to raising awareness of this growing women’s health crisis and plans to initiate a rigorous Phase 2b FREEDOM trial, with the first patient expected to be enrolled by the

end of 2020. The double-blind, randomized, placebo-controlled Phase 2b trial will evaluate the efficacy and safety of delivering autologous CD34+ cells (CLBS16) in subjects with CMD and without obstructive coronary artery disease.
CLBS119 for the repair of COVID-19-induced lung damage in COVID-19 survivors

Caladrius is committed to helping patients and communities combat the public health crisis of COVID-19 by leveraging its proprietary CD34+ cell technology to potentially repair COVID-19-induced lung damage. COVID-19 appears to damage the vasculature of the lungs and Caladrius believes the repair of that vasculature will prove necessary for patients to achieve a full recovery. Experience to date indicates that a large portion of COVID-19 survivors who required ventilatory support will suffer long-term, debilitating lung damage. While many developmental therapies responding to the COVID-19 pandemic are appropriately targeting the SARS-CoV-2 virus itself, or the manifestations of the acute phase of the illness, Caladrius is unaware of a therapy that has demonstrated the ability to repair COVID-19-induced lung damage. With consistent clinical and pre-clinical evidence that CD34+ cells can repair multiple organs, including models of severe lung inflammation, the Company sought and received FDA authorization for its investigational new drug ("IND") application for the study of CLBS119, a CD34+ cell therapy for the repair of COVID-19-induced lung damage. The planned 10-12-patient open-label, proof-of-concept clinical trial, is designed to evaluate the safety and efficacy of a single administration of CLBS119 for the treatment and repair of COVID-19-induced lung damage in adults. The study was recently initiated and patients who are experiencing hypoxia due to prior infection with SARS-CoV-2 and who require supplemental oxygen are now being screened for participation at NYU Langone Health.

Secures new capital to support cash runway through the end of 2021

As previously disclosed, in July 2020, Caladrius raised $2.0 million in a private placement priced at the market under Nasdaq rules. Caladrius has now successfully raised approximately $30 million in net proceeds year-to-date in 2020.

Third Quarter 2020 Financial Highlights

Research and development expenses were approximately $3.0 million for both the three months ended September 30, 2020 and the three months ended September 30, 2019. Research and development in both periods focused on the advancement of our ischemic repair platform. More specifically, R&D expense comprised (i) costs associated with investigational new drug application and planning for commencement of a pilot study of CLBS119, (ii) execution expenses for our ongoing registration-eligible study for CLBS12 in critical limb ischemia in Japan, and (iii) expenses for both the completion of our ESCaPE-CMD study of CLBS16 in coronary microvascular dysfunction and planning for the follow on Phase 2b study.

General and administrative expenses were approximately $2.3 million for the three months ended September 30, 2020, compared to $2.1 million for the three months ended September 30, 2019, representing an increase of 12%.

Overall, net losses were $5.3 million for the three months ended September 30, 2020, compared to $4.9 million for the three months ended September 30, 2019.

Balance Sheet Highlights

As of September 30, 2020, Caladrius had cash, cash equivalents and marketable securities of $40.3 million. Based on existing programs and projections, the Company remains confident that its current cash balances will fund its operations through 2021.

Conference Call

Caladrius will hold a conference call on Thursday, November 5, 2020, at 4:30 p.m. ET to discuss the financial results, provide a business update and answer questions. To join the conference call, please refer to the dial-in information provided below. The conference call will also be webcast live under the Investors section on the Company’s website at www.caladrius.com.

Cardiff Oncology Announces Third Quarter 2020 Results and Highlights

On November 5, 2020 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company developing drugs to treat cancers with the greatest medical need for new treatment options, including KRAS-mutated colorectal cancer, castration-resistant prostate cancer and leukemia, reported company highlights and financial results for the third quarter ended September 30, 2020. The Company is issuing this press release in lieu of conducting a conference call (Press release, Cardiff Oncology, NOV 5, 2020, View Source [SID1234570067]).

"Cardiff Oncology has seen robust growth over the past few months, which has been driven primarily by clinical data sets demonstrating safety and efficacy of onvansertib in solid tumor indications," said Dr. Mark Erlander, chief executive officer of Cardiff Oncology. "Data from our lead KRAS-mutated metastatic colorectal cancer (mCRC) program presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) conference, shows that the addition of onvansertib to standard-of-care therapy has led to durable responses and a significant improvement in the objective response rate (ORR) versus historical ORR’s of standard-of-care alone, highlighting onvansertib’s ability to address a critical unmet need in second-line treatment. We have also seen progress in our metastatic castration-resistant prostate cancer (mCRPC) program, presenting positive biomarker and efficacy data demonstrating onvansertib’s ability to overcome Zytiga resistance across known androgen receptor resistance mechanisms, at the Prostate Cancer Foundation (PCF) Scientific Retreat."

Dr. Erlander continued, "alongside our recent clinical achievements, we also executed on a major corporate milestone in early October with the Company closing an underwritten public offering of its common stock for gross proceeds of approximately $100 million. These funds will enable us to continue executing on our current programs and also initiate new clinical programs in other cancer indications."

Program highlights for the quarter ended September 30, 2020 include:

KRAS-mutated Metastatic Colorectal Cancer (mCRC) Program:

Presented clinical data at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 confirming the efficacy of onvansertib and the durability of response in second line KRAS-mutated mCRC patients

Newly announced data from a Phase 1b/2 clinical trial evaluating onvansertib in combination with FOLFIRI/bevacizumab in second line KRAS-mutated mCRC were featured in an electronic poster at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020. These data further demonstrate the safety, efficacy and durability of response of onvansertib in combination with FOLFIRI/bevacizumab and suggest that changes in plasma KRAS mutation levels may serve as a predictive biomarker of patient response. Data highlights from the presentation included:
•10 of 11 (91%) patients achieved disease control (SD – stable disease plus PR – partial response), with only 1 patient progressing in <6 months while on treatment
•5 of 11 (45%) patients achieved a PR; 4 patients had a confirmed PR with 1 patient going on to curative surgery; 1 patient with an initial PR went off study prior to confirmatory scan due to a non-treatment related event
8 of 11 (73%) patients demonstrated durable response ranging from 6 to >12 months, and 4 patients remain on treatment
•Patients achieving a PR showed the greatest decreases in plasma KRAS mutation levels (ranging from -78% to -100%) after one cycle of therapy
•Data demonstrated that onvansertib in combination with FOLFIRI/bevacizumab is safe and well tolerated

Hosted a Key Opinion Leader (KOL) call discussing KRAS-mutated colorectal cancer and highlighting data from the onvansertib Phase 1b/2 trial

The call featured KOLs Afsaneh Barzi, M.D., Ph.D. (City of Hope Comprehensive Cancer Center) and Heinz-Josef Lenz, M.D., FACP (USC Norris Comprehensive Cancer Center). In addition to a discussion of the latest data from the Phase 1b/2 trial of onvansertib in KRAS-mutated mCRC, the call also included an overview of the history of KRAS in clinical practice, the challenges of drug development and targeting of KRAS and the value of KRAS as a biomarker for patient selection and predicting response to treatment. You may access a replay of the event by clicking here.

Highlights for the period subsequent to the quarter end include:

Corporate Milestones:

Strengthened balance sheet with gross proceeds of approximately $100 million in offering of common stock

On October 2nd, Cardiff Oncology closed an underwritten offering of 6,500,000 shares of its common stock at a public offering price of $13.50 per share, before deducting underwriter discounts and commissions and estimated offering expenses. The underwriters also exercised an option to purchase an additional 975,000 shares at the public offering price (less underwriting discounts and commissions). Cardiff Oncology intends to use the net proceeds from this offering for clinical development of onvansertib, working capital and for other general corporate purposes.

Clinical Milestones:

Metastatic Castration Resistant Prostate Cancer (mCRPC) Program:

Presented positive efficacy and biomarker data from Phase 2 mCRPC trial demonstrating ability of onvansertib to overcome Zytiga resistance at the 27th Prostate Cancer Foundation (PCF) Scientific Retreat
An electronic poster presented at the PCF retreat featured new data and analyses related to an ongoing Phase 2 trial evaluating onvansertib in combination with Zytiga (abiraterone acetate) and prednisone in mCRPC patients. The poster included efficacy data demonstrating success in achieving the primary endpoint of disease control in patients showing initial resistance to Zytiga; safety across three different dose and dosing schedules, as well as the potential clinical benefit for patients with the basal molecular tumor subtype. Additional highlights from the presentation included:
•8 of 26 (31%) evaluable patients achieved the primary endpoint of disease control (defined by a lack of prostate specific antigen progression) after 12 weeks of treatment
•14 of 26 (54%) evaluable patients had stable disease (SD) after 12 weeks of treatment
•8 of 26 (31%) evaluable patients had a durable SD (>7 months)
Of 8 patients harboring androgen receptor alterations associated with Zytiga resistance, 3 achieved disease control at 12 weeks, 4 had SD at 12 weeks and 3 had a durable SD (>7 months)
•Median time on treatment was 9.2 months for patients with a decrease in circulating tumor cell (CTC) count (n=5) vs. 4.9 months for patients with a CTC increase (n=5)
•Genetic analyses identified a gene signature (biomarker) associated with onvansertib and Zytiga synergy in prostate cancer cells that is significantly enriched in the basal molecular subtype of prostate cancer patients
•Data shows that the combination of onvansertib and Zytiga is safe across all evaluated dosing schedules

Third Quarter 2020 Financial Results:

As of September 30, 2020, Cardiff Oncology had approximately $36.4 million in cash and cash equivalents.

Total operating expenses were approximately $4.5 million for the three months ended September 30, 2020, an increase of $0.2 million from $4.3 million for the same period in 2019. The increase in operating expenses is attributed to an increase in costs associated with clinical programs and outside services, stock-based compensation and staff costs.

Net cash used in operating activities in the third quarter of 2020 was $3.5 million, an increase of $0.3 million from $3.2 million for the same period in 2019. The increase is attributed to increases in operating expenses and the net changes in our operating assets and liabilities.

Research and development expenses increased by approximately $0.1 million to $2.9 million for the three months ended September 30, 2020, from $2.8 million for the same period in 2019. The increase in research and development expenses was primarily due to expenses associated with clinical programs and outside services.

Selling, general and administrative expenses increased by approximately $0.2 million to $1.6 million for the three months ended September 30, 2020 from $1.4 million for the same period in 2019. The increase is primarily due to an increase in stock-based compensation, staff costs and facilities, off-set by a decrease in outside services.

Y-mAbs Announces Third Quarter 2020 Financial Results and Recent Corporate Developments

On November 5, 2020 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a development-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the third quarter 2020 (Press release, Y-mAbs Therapeutics, NOV 5, 2020, View Source [SID1234570066]).

"We are very pleased with our third quarter 2020 financial results, especially seen in conjunction with the upcoming
PDUFA date for naxitamab later this month, and the planned resubmission of the omburtamab BLA. We believe that we are well positioned to transform Y-mAbs to a commercial-stage company," stated Thomas Gad, founder, Chairman and President.

Dr. Claus Moller, Chief Executive Officer, continued, "We are making good progress on the omburtamab BLA resubmission, and concurrently we’ve continued to advance many of the earlier stage programs in our pipeline. Nivatrotamab, our leading bispecific antibody, recently received ODD and RPDD from the FDA and our two INDs for 177Lu-omburtamab-DTPA in medulloblastoma and B7-H3 positive CNS/leptomeningeal metastasis in adults were recently cleared by the FDA."

Third Quarter 2020 and Recent Corporate Developments

Subsequent to the end of the third quarter, on October 26, 2020 Y-mAbs announced that the FDA has cleared the Company’s IND for 177Lu-omburtamab-DTPA for the treatment of B7-H3 positive CNS and Leptomeningeal Metastasis from tumors in adult patients

Also subsequent to the end of the third quarter, on October 16, 2020, Y-mAbs announced updates on naxitamab and omburtamab data, which were presented at the International Society of Pediatric Oncology conference

Also subsequent to the end of the third quarter, on October 14, 2020 Y-mAbs announced that the FDA has cleared the Company’s Investigational New Drug application for 177Lu-omburtamab-DTPA for the treatment of medulloblastoma, which is the most common type of primary brain cancer in children

Also subsequent to the end of the third quarter, on October 7, 2020, Y-mAbs announced that the FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation for its leading bispecific antibody product candidate nivatrotamab for the treatment of neuroblastoma

After the close of the third quarter, on October 5, 2020, Y-mAbs announced that it had received a Refusal to File letter from the FDA for the omburtamab BLA for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma. Subsequently, Y-mAbs requested and received what it believes to have been a positive Type A meeting with the FDA, and plans to work in close dialog with the Agency to amend the BLA with the goal of resubmitting by the end of 2020 or in early 2021. The BLA was originally submitted in August 2020

On July 14, 2020, Y-mAbs announced an update on the SADA technology and presented B7-H3 as a new preclinical SADA construct with potential use in prostate cancer
Financial Results

Y-mAbs reported a net loss of $32.8 million, or ($0.82) per basic and diluted share, for the three months ended September 30, 2020, compared to a net loss of $23.9 million, or ($0.70) per basic and diluted share, reported for the three months ended September 30, 2019.

For the nine months ended September 30, 2020, Y-mAbs reported a net loss of $99.4 million, or ($2.49) per basic and diluted share, compared to the net loss of $57.9 million, or ($1.69) per basic and diluted share, reported for the nine months ended September 30, 2019.

Operating Expenses

Research and Development
Research and development expenses were $21.0 million for the three months ended September 30, 2020, compared to $19.7 million for the three months ended September 30, 2019, an increase of $1.3 million. The increase in research and development expenses primarily reflects the following:

$2.4 million increase in personnel costs;
$0.5 million increase in clinical trial expenses;
$0.4 million increase in professional and consulting fees; and
$2.0 million offsetting decrease in outsourced manufacturing cost
Research and development expenses were $69.7 million for the nine months ended September 30, 2020, compared to $46.7 million for the nine months ended September 30, 2019, an increase of $23.0 million. The increase in research and development expenses primarily reflects the following:

$13.3 million increase in milestones and license fees related to the SADA upfront cash payment and stock issuances and accrued milestones;
$6.3 million increase in personnel costs; and
$1.9 million increase in outsourced research and supplies to support the expansion of our product development activities
General and Administration
General and administrative expenses were $11.6 million for the three months ended September 30, 2020, compared to $4.7 million for the three months ended September 30, 2019, an increase of $6.9 million. Such increase in general and administrative expenses primarily reflects the following:

$3.2 million increase in commercial infrastructure costs;
$2.2 million increase in personnel costs; and
$1.6 million increase in business insurance and professional fees
General and administrative expenses were $30.2 million for the nine months ended September 30, 2020, compared to $12.6 million for the nine months ended September 30, 2019, an increase of $17.6 million. Such increase in general and administrative expenses primarily reflects the following:

$8.7 million increase in commercial infrastructure costs;
$5.8 million increase in personnel costs; and
$3.1 million increase in business insurance and professional fees
Cash and Cash Equivalents

The Company had approximately $131.3 million in cash and cash equivalents as of September 30, 2020

Webcast and Conference Call

The Company will host a conference call on Friday, November 6, 2020 at 9 a.m. Eastern Time. To participate in the call, please dial 877-407-0792 (domestic) or 201-689-8263 (international) and reference the access code 13712633. A webcast will be available at: View Source

APOLLO ENDOSURGERY, INC. REPORTS THIRD QUARTER 2020 RESULTS

On November 5, 2020 Apollo Endosurgery, Inc. ("Apollo") (Nasdaq: APEN), a global leader in less invasive medical devices for gastrointestinal and bariatric procedures, reported financial results for the third quarter ended September 30, 2020 (Press release, Apollo Endosurgery, NOV 5, 2020, View Source [SID1234570065]).
Third Quarter 2020 and Recent Highlights
•Total Endoscopy revenue increased 21% compared to the third quarter of 2019 to $12.5 million
•U.S. Endoscopy revenue increased 37% compared to the third quarter of 2019 to $6.6 million
•Gross margin increased to 55% compared to 48% in the third quarter of 2019
•Net loss improved by 70% to $2.6 million compared to $8.7 million for the third quarter of 2019
•Ended the quarter with $38.2 million in cash, cash equivalents and restricted cash
"The third quarter rebound in elective procedures in our direct markets was both quicker and stronger than our early expectations," stated Todd Newton, Apollo’s Chief Executive Officer. "We also took steps in the third quarter to lower our annual operating expenses and improve our liquidity position. As a result, we believe our liquidity provides sufficient runway through 2021 and through the COVID-19 pandemic, completion of the MERIT trial, and launch of X-Tack."
Third Quarter Results
Worldwide Endoscopy product sales were $12.5 million for the third quarter of 2020, an increase of 21% compared to the third quarter of 2019 as the easing of public health interventions due to the COVID-19 pandemic generated a resurgence in elective procedures that use our Endoscopy products.
Geographically, U.S. Endoscopy sales increased 37% to $6.6 million for the third quarter of 2020 and OUS Endoscopy sales increased 7% to $5.9 million.
By product group, U.S. ESS product sales increased 28% to $4.7 million for the third quarter of 2020 compared to the same period in 2019, while OUS ESS sales remained unchanged at $2.9 million. U.S. IGB product sales increased 70% to $1.9 million for the third quarter of 2020, while OUS IGB product sales increased 15% to $3.0 million.
Total revenue for the third quarter of 2020 was $12.8 million, an increase of 14% from $11.3 million in the prior year third quarter. Excluding $0.1 million and $0.8 million of transition service revenue in the third quarter of 2020 and 2019, respectively, related to the Surgical product line which was divested in December of 2018, total revenue increased 21%.
Gross margin increased to 55% for the third quarter of 2020 from 48% in the third quarter of 2019 primarily due to benefits from completed gross margin improvement projects and the higher mix of IGB product sales and direct market sales as a percentage of total revenue.
Total operating expenses decreased $3.7 million, or 30%, for the third quarter of 2020 due to the liquidity preservation program implemented in the second quarter of 2020 in response to the COVID-19 pandemic.
Net loss for the third quarter of 2020 was $2.6 million compared to $8.7 million for the third quarter of 2019, an improvement of 70%.
Cash, cash equivalents and restricted cash were $38.2 million as of September 30, 2020.
Conference Call
Apollo will host a conference call on November 5, 2020 at 3:30 p.m. Central Time / 4:30 p.m. Eastern Time to discuss operating results for the third quarter ended September 30, 2020. To join the conference call by telephone, please dial +1-785-424-1667. A live webcast of the conference call will be made available on the "Events and Presentations" section of our Investor Relations website: ir.apolloendo.com.
A replay of the webcast will be made available on Apollo’s website, www.apolloendo.com following the event.

Regulus Therapeutics Reports Third Quarter 2020 Financial Results and Recent Updates

On November 5, 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 third quarter ended September 30, 2020 and provided a corporate update (Press release, Regulus, NOV 5, 2020, View Source [SID1234570064]).

"We are pleased with the progress on our ADPKD program with the recent announcement of dosing of the first patient in the Phase 1b clinical trial of RGLS4326 in patients with ADPKD", stated Jay Hagan, CEO of Regulus. "Next year is shaping up to be an important one for Regulus, with data from the first cohort anticipated by end of Q1 2021. We also achieved $10 million in milestones from our collaboration with Sanofi enabling us to reduce our outstanding debt to $4.7 million and extend the interest-only period through the end of 2021."

Third Quarter 2020 Corporate Highlights and Recent Updates

Amendment of Sanofi Collaboration Agreement and Achievement of Milestones: In August 2020, the Company entered into an amendment with Sanofi concerning the receipt of potential milestones from Sanofi for its development of miR-21 programs. Under the terms of the amendment with Sanofi, the Company was eligible to receive $4 million upon the completion of transfer and verification of certain materials valued at an additional $1 million sold to Sanofi. In addition to this payment of $5 million received, the Company achieved a $5 million interim enrollment milestone and is eligible to receive an additional $25 million upon the successful achievement of a development milestone related to the Phase 2 HERA study.

Term Loan Amendments and Reduction in Principal: In August 2020, and concurrently with the Sanofi amendment, the Company amended its term loan agreement with Oxford LLC, under which the Company is eligible for up to an additional seven months of interest only payments in the event the Company pays down $10 million in loan principal before April 30, 2021. In September and October 2020, the Company made payments totaling $5 million to Oxford and plans to make an additional $5 million payment to Oxford before the end of 2020, thus triggering the additional interest only period. These payments will reduce the remaining principal due under the term loan to approximately $4.7 million. Remaining principal and accrued interest payments will commence on January 1, 2022.

Program Updates

RGLS4326 for ADPKD: In October 2020, the Company dosed the first patient in a Phase 1b clinical study for RGLS4326 in patients with ADPKD. The Phase 1b is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD and will evaluate administration of RGLS4326 for safety, pharmacokinetics, and changes in levels of polycystin 1 (PC1) and polycystin 2 (PC2). Patients with ADPKD, due to a mutation in the PKD genes, have been reported to have low levels of PC1 and PC2, the proteins encoded by the PKD1 and PKD2 genes, respectively. This study is designed to assess whether different dose levels of RGLS4326 can increase levels of PC1 and PC2 in ADPKD patients. The first cohort is planned to enroll up to nine patients who will receive RGLS4326 every two weeks over a six week period. The Company anticipates availability of results from the first cohort by the end of Q1 2021.
Advancement of Hepatitis B virus (HBV) Program: The Company has identified several microRNA targets that serve as host factors for the hepatitis B virus (HBV). Our lead compound directed to one of the host microRNAs has demonstrated nanomolar potency against HBV DNA replication and more than 95% reduction in Hepatitis B surface antigen in in vitro studies. Additionally, we have demonstrated reduction of HBV DNA, surface antigen and pgRNA in an in vivo efficacy model. The Company believes that targeting a host factor in the liver represents a unique mechanism of action for treatment of the virus compared to other programs in development and holds the potential for achieving a functional cure. The Company has nominated a development candidate and plans to commence IND-enabling activities.
Additional Research Updates: Cell therapies have been approved to treat a variety of hematological malignancies. Targeting solid tumors, however, has proven challenging for cell therapies due to the immune-suppressive effect of the tumor microenvironment (TME). The Company believes that ex vivo modulation of microRNA may enable cell therapy approaches to overcome the effects of the TME and address other challenges faced by cell therapies. The Company has demonstrated that targeting microRNA ex vivo can improve certain characteristics of engineered cells including improved in vitro expansion, effector function, cytokine production, as well as resistance to exhaustion induced by tonic signaling. The Company is pursuing multiple applications of microRNA technology in a variety of cell therapies.
Third Quarter 2020 Financial Results

Cash Position: As of September 30, 2020, Regulus had $17.8 million in cash and cash equivalents.

Revenue: Revenue was $5.0 million for the three and nine months ended September 30, 2020, respectively, compared to less than $0.1 million and $6.8 million for the three and nine months ended September 30, 2019, respectively. Revenue recognized for the three and nine months ended September 30, 2020 was attributable to the completion of transfer and verification of certain materials to Sanofi under the August 2020 amendment. Revenue recognized for the nine months ended September 30, 2019 was attributable to the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Research and Development (R&D) Expenses: Research and development expenses were $4.0 million and $11.4 million for the three and nine months ended September 30, 2020, respectively, compared to $2.4 million and $10.3 million for the three and nine months ended September 30, 2019, respectively. The aggregate increase for the three months ended September 30, 2020, as compared to the three months ended September 30, 2019, was driven by an increase in external development expenses, primarily attributable to FDA lifting the partial hold on the MAD study in December 2019 and completion of the dosing for the Phase 1 of the MAD study in July 2020, as well as start-up activities leading up to the first patient dosing in our RGLS4326 Phase 1b study in October 2020. The aggregate increase for the nine months ended September 30, 2020, as compared to the nine months ended September 30, 2019, was driven by an increase in external development expenses, attributable to RGLS4326 MAD and Phase 1b study activities, partially offset by a reduction in personnel and internal expenses.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.1 million and $6.7 million for the three and nine months ended September 30, 2020, compared to $2.6 million and $9.0 million for the three and nine months ended September 30, 2019. These amounts reflect personnel-related and ongoing general business operating costs. The decreases for the three and nine months ended September 30, 2020, as compared to the three and nine months ended September 30, 2019, are primarily attributable to continued cost reduction efforts subsequent to our corporate restructurings.

Net Loss: Net loss was $1.5 million, or $0.04 per share (basic and diluted), and $14.4 million, or $0.47 per share (basic and diluted), for the three and nine months ended September 30, 2020, respectively, compared to $5.4 million, or $0.26 per share (basic and diluted), and $13.7 million, or $0.86 per share (basic and diluted), for the same periods 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, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by FDA until the second set of requirements outlined by the agency have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from the recently completed additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration. RGLS4326 received orphan drug designation from FDA in July 2020.