On November 10, 2021 Schrödinger, Inc. (Nasdaq: SDGR), whose physics-based software platform is transforming the way therapeutics and materials are discovered, reported financial results for the third quarter ended September 30, 2021, and provided an update on the company (Press release, Schrodinger, NOV 10, 2021, View Source [SID1234595096]).

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"We are very pleased with the progress we have made across the business so far this year. The level of engagement with senior R&D leaders across pharma and the growth we are seeing in new software customers suggests a shift in the drug discovery paradigm and a push to incorporate advanced computational approaches into projects at all stages," stated Ramy Farid, Ph.D., chief executive officer at Schrödinger. "As we approach year-end, we are continuing to focus on key strategic priorities, including advancing our internal pipeline. We are on track to submit an IND to the FDA for our MALT1 inhibitor program in the first half of next year, and we look forward to presenting additional preclinical data for this program at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting next month. We also recently selected a development candidate and initiated IND-enabling studies for our CDC7 inhibitor program, and we added a new oncology target, our fifth internal program, to our pipeline."

Recent Business Highlights
Collaborations Highlight Opportunities and Progress Across the Business

Entered collaboration with Centessa Pharmaceuticals Subsidiary, Orexia Therapeutics, focused on novel orexin receptor agonists: In October, Schrödinger and Centesa Pharmaceuticals Subsidiary, Orexia Therapeutics, announced a collaboration focused on the discovery of novel therapeutics targeting the orexin-2 receptor, which is known to play a role in a broad spectrum of sleep disorders, including narcolepsy. The collaboration provides Orexia with substantial access to Schrödinger’s entire computational platform as well as Schrödinger’s extensive expertise in ultra-large-scale deployment of its technology.

Under the terms of the agreement, Orexia is responsible for preclinical research activities, clinical development and commercialization of future product candidates discovered under the collaboration. Schrödinger received an upfront software access payment and may become eligible to receive certain preclinical, development, regulatory and commercial milestone payments, as well as low single digit royalties on global net sales.
Formed strategic collaboration with MD Anderson to accelerate development of WEE1 program: In October, Schrödinger and The University of Texas MD Anderson Cancer Center announced a two-year strategic research collaboration focused on accelerating and optimizing the development of Schrödinger’s WEE1 inhibitor program. The goal of the collaboration is to accelerate and optimize the clinical development path for Schrödinger’s WEE1 program through molecular biomarker-driven tumor type prioritization and patient stratification and to validate biomarkers to predict response or resistance to a WEE1 inhibitor. The joint team will seek to prioritize clinical studies of a WEE1 inhibitor as a single agent in selected cancer indications and in combinations for defined clinical subpopulations.
Completion of $100 million financing by strategic partner ShouTi: In October, Schrödinger’s partner, ShoutTi Inc., announced a $100 million series B financing to advance the company’s discovery platform, leveraging Schrödinger’s computational platform and expertise, and ShouTi’s structural biology and drug discovery expertise to design orally available medicines with properties that aim to overcome current limitations of biologic and peptide drugs. Schrödinger is a co-founder of ShouTi, which is advancing a pipeline focused on chronic diseases with high medical need, including cardiovascular, metabolic and pulmonary conditions.
Provided update on collaboration with Bristol Myers Squibb: Schrödinger reported that the companies have prioritized an undisclosed precision oncology target that will replace HIF-2 alpha. Additionally, Schrödinger and Bristol Myers Squibb recently expanded their collaboration with a new agreement to discover, develop and commercialize bifunctional degraders.
Internal Programs Expanding and Advancing

New preclinical data from multiple MALT1 inhibitors to be presented at ASH (Free ASH Whitepaper): Last week, Schrödinger announced that new preclinical data from its MALT1 program will be presented at the ASH (Free ASH Whitepaper) 2021 Annual Meeting, which is being held December 11 – 14 virtually and in person in Atlanta, GA. The poster presentation, "Characterization of Potent Paracaspase MALT1 Inhibitors for Hematological Malignancies" (Abstract #1187), is scheduled to take place on Saturday, December 11, from 5:30 p.m. – 7:30 p.m. ET. MALT1 is considered a potential therapeutic target for several non-Hodgkin’s B-cell lymphomas as well as chronic lymphocytic leukemia.

Schrödinger expects to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration for its MALT1 program in the first half of 2022.
Development candidate selected for CDC7 program: Schrödinger reported that it selected a development candidate and has initiated IND-enabling studies for its CDC7 inhibitor program. CDC7 is thought to be linked to cancer cells’ proliferative capacity and ability to bypass normal DNA damage responses. Targeting proteins that play important roles in DNA replication and replication stress, such as CDC7, is gaining momentum as a therapeutic approach for cancer.
Internal pipeline expanded to five programs: Schrödinger continued to advance its discovery efforts and reported that the company further expanded its internal pipeline with the addition of a fifth program, which is an undisclosed oncology target.
Third Quarter 2021 Financial Results

Total revenue was $29.9 million for the third quarter of 2021, a 16 percent increase compared to the third quarter of 2020.
Software revenue was $24.3 million for the third quarter of 2021, compared to $22.9 million for the third quarter of 2020. The six percent growth observed year-over-year was primarily due to increased sales from existing customers and the addition of new customers, partially offset by multi-year contracts that were executed in the third quarter of 2020, as well as the completion of a research project that was active in the third quarter of 2020.
Drug discovery revenue was $5.6 million for the third quarter of 2021, compared to $2.9 million in the third quarter of 2020. Third quarter 2021 drug discovery revenue included the recognition of $4.4 million of revenue from the company’s collaboration with Bristol Myers Squibb.
Gross profit was $11.1 million in the third quarter of 2021, compared to $15.3 million in the third quarter in 2020. Software gross margin was 73 percent in the third quarter of 2021, compared to 81 percent for the same period in the prior year, reflecting planned investments to drive and support large-scale adoption of Schrödinger’s platform as well as increased royalty expenses in the quarter.
Operating expenses for the third quarter of 2021 were $45.8 million, compared to $30.7 million in the third quarter of 2020, driven by expenses required to scale the company’s business, advance its internal drug discovery programs and continue to build a public company infrastructure.
Other expense, which included changes in fair value of equity investments and interest income, was $0.3 million in the third quarter of 2021 compared to income of $18.7 million for the third quarter of 2020 due to adjustments to the fair value of the company’s equity investments.
Net loss, after adjusting for non-controlling interest, was $35.0 million for the third quarter of 2021, compared to net income of $3.9 million for the third quarter of 2020, driven by adjustments to the fair value of the company’s equity investments as well as planned investments to advance the company’s growth strategy.
Cash, cash equivalents, restricted cash and marketable securities as of September 30, 2021, were $600.2 million, compared to $616.6 million as of June 30, 2021.
Full-Year 2021 Financial Outlook

As of November 10, 2021, Schrödinger expects total revenue to range from $124 million to $134 million for the fiscal year ending December 31, 2021. The company is maintaining its full-year software revenue expectation of $102 million to $110 million. The company is adjusting its full-year drug discovery revenue expectation to a range of $22 million to $24 million, updated from a previous expectation of $22 million to $32 million, primarily due to the timing of anticipated milestones from collaborators.

Schrödinger continues to aggressively fund R&D to advance its technology and drug discovery pipeline. The company continues to expect operating expense growth to be higher than the 42 percent annual growth rate reported in 2020 and expects software gross margin to be lower than the 81 percent reported in 2020.

Webcast and Conference Call Information

Schrödinger will host a conference call to discuss its third quarter financial results on Wednesday, November 10, 2021, at 4:30 p.m. ET. The conference call can be accessed live by dialing (833) 727-9520 (domestic) or +1 (830) 213-7697 (international) and referring to conference ID 2484045. The webcast can also be accessed under "News & Events" in the investors section of Schrödinger’s website, View Source The archived webcast will be available on Schrödinger’s website for approximately 90 days following the event.

On November 10, 2021 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company developing itolizumab to treat severe autoimmune and inflammatory disorders with high unmet medical need, reported financial results for the third quarter 2021, and provided an update on its clinical programs (Press release, Equillium, NOV 10, 2021, View Source [SID1234595095]).

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"Following positive topline data from the EQUATE study, we started the third quarter by announcing our plans to advance itolizumab into a pivotal clinical study in first-line acute graft-versus-host disease, which we anticipate commencing early in the new year," said Bruce Steel, chief executive officer at Equillium. "Recent strategic activity in the GVHD therapeutic segment demonstrates industry’s recognition of the high unmet medical need for effective treatments for patients suffering from this deadly disease, and we believe our pivotal study could position itolizumab to become the first approved therapy to treat patients with acute GVHD in the first-line setting. We are also encouraged by the reductions in proteinuria observed in the subgroup data from the Type A portion of the EQUALISE study in patients with systemic lupus erythematosus, without lupus nephritis, that had elevated baseline urine protein/creatinine and albumin/creatinine ratios. Based on the Type A data we amended the protocol in the Type B portion to include newly diagnosed patients in addition to refractory patients and selected the 1.6 mg/kg dose. As a result of these protocol enhancements and due to challenges in patient recruitment amid the ongoing global pandemic, we now expect to announce interim data from the Type B portion of the study in patients with lupus nephritis mid-year 2022. We look forward to initiating our pivotal study in first-line acute GVHD early in the new year and presenting topline data from our EQUIP study in moderate to severe asthma before the end of this year."

Corporate & Clinical Highlights Since Beginning of Q3 2021:

Announced plans to initiate a pivotal study in first-line acute graft-versus-host disease (aGVHD) following positive topline results from the EQUATE study and an End-of-Phase 1 meeting with the FDA
Reported additional data from the EQUALISE study’s Type A systemic lupus erythematosus (SLE) patients, without lupus nephritis, that had elevated baseline urine protein/creatinine and albumin/creatinine ratios, demonstrating a mean decrease of 42% and 54%, respectively, at Day 57 following two doses of itolizumab
Presented multiple posters at the American College of Rheumatology (ACR), the American Society of Nephrology (ASN) and the American College of Allergy, Asthma and Immunology (ACAAI)
ASN & ACR
Sustained decrease in proteinuria observed in subgroup of SLE patients, without lupus nephritis, following two doses of itolizumab
Itolizumab was safe and well tolerated at doses ranging from 0.4 to 2.4 mg/kg
Dose dependent decreases in inflammatory marker CD6 following itolizumab administration
ACAAI
◦ Itolizumab was well tolerated in patients with moderate to severe uncontrolled asthma

Upcoming Catalysts:

EQUIP Phase 1b study: topline data in uncontrolled asthma expected Q4 2021
Initiate pivotal study in first-line aGVHD expected early 2022
EQUALISE Phase 1b study: interim data from Type B lupus nephritis patients expected mid-2022
Upcoming Catalysts:

EQUIP Phase 1b study: topline data in uncontrolled asthma expected Q4 2021
Initiate pivotal study in first-line aGVHD expected early 2022
EQUALISE Phase 1b study: interim data from Type B lupus nephritis patients expected mid-2022
Third Quarter 2021 Financial Results

Research and development (R&D) expenses for the third quarter of 2021 were $7.0 million, compared with $4.2 million for the same period in 2020. The increase was driven by greater clinical development expenses related to EQUIP primarily resulting from a lower R&D Tax Incentive benefit from the Australian Taxation Office and by start-up expenses related to our planned pivotal study in aGVHD, partially offset by lower costs related to a Phase 3 COVID-19 clinical trial that we decided not to commence. Greater employee compensation and benefit expenses driven by increased headcount and greater overhead expenses and costs related to our non-clinical research activities also contributed to the increase in R&D expense.

General and administrative (G&A) expenses for the third quarter of 2021 were $2.9 million, compared with $2.3 million for the same period in 2020. The increase was driven by greater employee compensation and benefit expenses primarily related to increased headcount and by greater overhead expenses, partially offset by a decrease in consulting expenses.

Net loss for the third quarter of 2021 was $10.3 million, or $(0.35) per basic and diluted share, compared with a net loss of $6.6 million, or $(0.31) per basic and diluted share for the same period in 2020. The increase in net loss was largely attributable to greater operating expenses.

Cash used in operations for the third quarter of 2021 was $7.0 million compared to $7.0 million in the second quarter of 2021.

Cash, cash equivalents and short-term investments totaled $90.7 million as of September 30, 2021, compared to $97.6 million as of June 30, 2021. Equillium believes that its cash and investments will be sufficient to fund its currently planned operations into 2023.

About Itolizumab

Itolizumab is a clinical-stage, first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM signaling pathway to selectively downregulate pathogenic T effector cells while preserving T regulatory cells critical for maintaining a balanced immune response. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited.

On November 10, 2021 Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, reported that it will present at the Jefferies London Healthcare Conference, which is taking place November 16-19, 2021 (Press release, Corvus Pharmaceuticals, NOV 10, 2021, View Source [SID1234595094]). The Company will conduct one-on-one meetings with investors at this conference and a pre-recorded corporate overview presentation by Richard A. Miller, M.D., president and chief executive officer of Corvus, will be available to play on-demand starting at 3:00 am ET on November 18.

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Attendees can register to view the webcast here. A webcast of the presentation will be available via the investor relations section of the Corvus website for 30 days following the event.

On November 10, 2021 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported financial and operating results for the third quarter 2021 (Press release, Lineage Cell Therapeutics, NOV 10, 2021, View Source [SID1234595093]). Lineage will host a conference call today at 4:30 p.m. Eastern Time to discuss its third quarter 2021 financial results and to provide a business update.

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"Lineage’s approach is to produce specific types of human cells and stably transplant those cells as a treatment for serious medical conditions. We believe our approach, in certain settings, can generate clinical outcomes beyond the reach of traditional methods, as evidenced by the restoration of retinal tissue in patients in our dry AMD trial and the restoration of a tissue matrix in patients in our spinal cord injury trial," stated Brian M. Culley, Lineage CEO. "During the third quarter, we reported positive interim outcomes in patients with dry AMD with geographic atrophy, initiated performance testing of our OPC1 delivery device for spinal cord injury, and we expanded our executive team with the appointment of a new General Counsel. Looking ahead, we are preparing for engagement with FDA for our OpRegen program to discuss aspects of product designation, manufacturing plans, and later-stage clinical development. In parallel, we look forward to the initiation of our OPC1 and novel delivery device clinical safety study early next year. We believe our technology platform has broad potential beyond even the indications we currently are pursuing and while we continue to advance our three clinical-stage programs, we also are evaluating new applications of our technology, either on our own or through strategic alliances."

Some of the milestones achieved in the third quarter include:

– Presented OpRegen clinical data at the 54th Annual Scientific Meeting of the American Retina Society from the ongoing Phase 1/2a study of OpRegen for the treatment of dry-AMD with GA; statistically significant evidence of a treatment effect with OpRegen was observed in Cohort 4 better vision patients.

– Reported continued positive interim clinical data with OpRegen: 8/12 (67%) of the Cohort 4 patients’ treated eyes were at or above baseline visual acuity at their last assessment, based on per protocol scheduled visits ranging from 9 months to over 3 years post-transplant, while visual acuity predictably declined in the majority of untreated eyes; notably, three patients with evidence of retinal restoration and confirmed history of GA growth continued to demonstrate areas of retinal restoration as of their last per protocol assessments, ranging from 9 months to 33 months following treatment.

– Announced the appointment of George A. Samuel III as General Counsel and Corporate Secretary. Mr. Samuel brings extensive corporate, transactional, intellectual property and commercial expertise which spans nearly 15 years across the life sciences and technology sectors as well as in private practice.

– Featured in the B. Riley Securities Fall 2021 "Growth Biotech Best Idea" Virtual Series as well as the 2021 Cantor Fitzgerald Virtual Global Healthcare Conference.

Some of the events and milestones anticipated by Lineage include:

– OpRegen Program

Additional interim data from the Phase 1/2a clinical study to be featured at the 2021 American Academy of Ophthalmology Annual Meeting in a presentation on November 13, 2021, as part of the Gene and Cell-Based Therapies Session, by Michael S. Ip, M.D., Professor, Department of Ophthalmology at the David Geffen School of Medicine at the University of California, Los Angeles.
Multiple interactions with the U.S. Food and Drug Administration (FDA) planned to discuss product designation, manufacturing plans, and later-stage clinical development, anticipated to begin in Q4 2021 and continue in Q1 2022.
– OPC1 Program

Complete evaluation of a novel Parenchymal Spinal Delivery (PSD) system in non-clinical testing; anticipated in Q4 2021.
Complete GMP production of OPC1 via an improved and larger-scale manufacturing process and a new thaw-and-inject formulation; anticipated in Q1 2022.
FDA interaction to discuss recent manufacturing improvements made to OPC1; anticipated in Q1 2022.
Initiation of clinical performance and safety testing of the novel PSD device for OPC1; anticipated Investigational New Drug (IND) amendment submission in Q1 2022.
– VAC Programs

Completion of enrollment by Cancer Research UK in the ongoing VAC2 Phase 1 non-small cell lung cancer study; anticipated in Q1 2022.
Continued development of a dendritic cell-based therapeutic for glioblastoma with our strategic partner; ongoing throughout 2022.
Evaluation of opportunities for new VAC product candidates based on internally identified or partnered tumor antigens; ongoing throughout 2022.
– Business Development

Evaluation of partnership opportunities and expansion of existing collaborations; ongoing throughout 2022.
Balance Sheet Highlights

Cash, cash equivalents, and marketable securities totaled $65.1 million as of September 30, 2021. Marketable securities of $4.3 million as of September 30, 2021 include the Company’s remaining ownership in OncoCyte Corporation ("OncoCyte") and Hadasit Bio-Holdings Ltd.

Third Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from research grants, royalties, and licensing fees. Total revenues for the three months ended September 30, 2021 were approximately $2.3 million, an increase of $1.7 million as compared to $0.6 million for the same period in 2020. The increase was primarily related to a $1.6 million increase in royalty revenues, and a $0.3 million increase in licensing revenues in connection with a collaboration agreement, partially offset by a $0.2 million decrease in grant revenues.

Operating Expenses: Operating expenses are comprised of research and development (R&D) expenses and general and administrative (G&A) expenses. Total operating expenses for the three months ended September 30, 2021 were $8.1 million, an increase of $0.9 million as compared to $7.2 million for the same period in 2020.

R&D Expenses: R&D expenses for the three months ended September 30, 2021 were $2.8 million, a decrease of approximately $0.8 million as compared to $3.6 million for the same period in 2020. The decrease was primarily driven by lower VAC program expenses, related to a non-recurring prior year accrual of a $1.6 million signature fee to Cancer Research UK, partially offset by an increase in OPC1 expenses resulting from a return of unspent project funds of approximately $0.8 million in the prior year period from a former Asterias BioTherapeutics, Inc. ("Asterias") service provider.

G&A Expenses: G&A expenses for the three months ended September 30, 2021 were $5.3 million, an increase of approximately $1.7 million as compared to $3.6 million for the same period in 2020. The increase was primarily attributable to increases of $0.8 million in litigation and other expenses related to Lineage’s merger with Asterias, and $0.5 million in share-based compensation.

Loss from Operations: Loss from operations for the three months ended September 30, 2021 was approximately $6.8 million, an increase of $0.1 million as compared to $6.7 million for the same period in 2020.

Other Income/(Expenses), Net: Other income/(expenses), net for the three months ended September 30, 2021 reflected other expense, net of ($2.0) million, compared to other expense, net of ($1.2) million for the same period in 2020. The variance was primarily related to a decrease in the value of Lineage’s OncoCyte shares, a decrease in interest income following settlement of the Juvenescence Limited note receivable in the prior year, and no sales of marketable equity securities as compared to the prior year’s quarter.

Net loss attributable to Lineage: The net loss attributable to Lineage for the three months ended September 30, 2021 was $7.8 million, or $0.05 per share (basic and diluted), compared to a net loss attributable to Lineage of $7.8 million, or $0.05 per share (basic and diluted), for the same period in 2020.

Conference Call and Webcast

Lineage will host a conference call and webcast today, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its third quarter 2021 financial results and to provide a business update. 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 November 18, 2021, 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 9352189.

On November 10, 2021 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, 2021 and provided a corporate update (Press release, Regulus, NOV 10, 2021, View Source [SID1234595092]).

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"We are pleased with the progress we have made in advancing our next generation candidate RGLS8429 as a potential treatment for ADPKD, as the compound has been shown to have the favorable properties of RGLS4326 without the limitations of the first-generation compound," commented Jay Hagan, CEO of Regulus Therapeutics. "RGLS8429 has demonstrated comparable potency, as well as similar pharmacokinetic and pharmacodynamic profiles, without the off-target CNS effects seen in chronic preclinical toxicology studies with RGLS4326 at the top doses tested. We look forward to our pre-IND meeting in December with the FDA to help finalize our IND submission and reach another important milestone in our mission to improve the lives of ADPKD patients."

Program Updates

RGLS8429 for ADPKD: In October 2021, the Company discontinued development of its first-generation compound, RGLS4326, to allocate resources and efforts towards the development of its more promising next-generation compound, RGLS8429. The Company believes RGLS8429 has demonstrated a superior pharmacological profile, with the absence of the off-target central nervous system (CNS) effects that were seen with RGLS4326 at the top doses tested in chronic preclinical toxicology studies. RGLS8429 has also shown equal potency to RGLS4326 for its molecular target (miR-17) in both in-vitro and in-vivo efficacy studies. The Company expects to have a pre-IND meeting with the U.S. Food and Drug Administration (FDA) for RGLS8429 before year-end and is on track for an IND submission and initiation of clinical development in the second quarter of 2022, subject to FDA clearance of the IND.

The Company’s Phase 1 plans include a single dose escalation study in healthy volunteers to enable a multi-dose escalation study in ADPKD patients around the dose levels where robust clinical biomarker effects were demonstrated with RGLS4326. The Company anticipates reporting top-line biomarker data in the first cohort of RGLS8429-treated patients in early 2023.

RGLS4326 for ADPKD: On November 4 and November 9, data from the first cohort of patients in the Phase 1b clinical trial of RGLS4326, the Company’s first-generation compound, for the treatment of ADPKD, was presented at the American Society of Nephrology (ASN) Kidney Week, and at the Biomarkers for Rare Diseases Summit. In the first cohort, nine patients were enrolled and received 1 mg/kg of RGLS4326 subcutaneously every other week for four doses. The mean increase in polycystins 1 and 2 at the end of study compared to baseline levels for all nine patients in the first cohort were 58% (p=.0004) and 38% (p=.026), respectively. These data demonstrate clinical evidence that treatment with RGLS4326 increased PC1 and PC2, most likely through inhibition of miR-17 in the kidney of patients with ADPKD. These results also imply that overexpressed miR-17 in ADPKD patients represses Pkd1 and Pkd2 expression, further validating miR-17 as a therapeutic target for ADPKD treatment. The details for each presentation are below:

ASN Kidney Week ePoster:
Poster Title: RGLS4326 Increases Urinary PC1 and PC2 Levels in Individuals with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Poster Date and Time: Thursday, November 4, 2021, 10:00 AM PDT
Poster Number: PO1244

Biomarkers for Rare Diseases Summit Presentation:
Presentation Title: Results from the First Cohort of Phase1b Clinical Trial of RGLS4326 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Presenter: Edmund Lee, PhD, Executive Director, Biology, Regulus Therapeutics
Presentation Date and Time: Tuesday, November 9, 2021, 9:30 AM PDT

A copy of each presentation is available at www.regulusrx.com/publications/

Financial Results

Cash Position: As of September 30, 2021, Regulus had $35.8 million in cash and cash equivalents.

Research and Development (R&D) Expenses: Research and development expenses were $5.9 million and $13.4 million for the three and nine months ended September 30, 2021, respectively, compared to $4.0 million and $11.4 million for the same periods in 2020, respectively. These amounts reflect internal and external costs associated with advancing our clinical and preclinical pipeline.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.5 million and $7.5 million for the three and nine months ended September 30, 2021, respectively, compared to $2.1 million and $6.7 million for the same periods in 2020, respectively. These amounts reflect personnel-related and ongoing general business operating costs.

Net Loss: Net loss was $8.6 million, or $0.10 per share (basic and diluted), and $20.7 million, or $0.26 per share (basic and diluted), for the three and nine months ended September 30, 2021, compared to $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 same periods in 2020.

Conference Call and Webcast Information:
The Company will host a conference call and live audio webcast today at 5:00 p.m. Eastern Daylight Time to discuss its third quarter 2021 financial results and corporate update. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 2108429. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode ID 2108429. The webcast and telephone replay will be archived on the Company’s website at www.regulusrx.com following the call.

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 RGLS8429

RGLS8429 is a novel, second generation oligonucleotide designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity, as well as a superior pharmacologic profile have been demonstrated. Regulus has nominated RGLS8429 as a development candidate for the treatment of ADPKD.