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.

On November 10, 2021 Heat Biologics, Inc. ("Heat") (NASDAQ: HTBX), reported that strategic, financial, and operational updates for the third quarter ended September 30, 2021 (Press release, Heat Biologics, NOV 10, 2021, View Source [SID1234595090]).

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Jeff Wolf, Chief Executive Officer of Heat, commented, "Our clinical and preclinical programs are progressing well. In September, we expanded dosing in our Phase 1 clinical trial of monoclonal antibody PTX-35 in patients with solid tumors, and are planning a clinical development pathway to utilize PTX-35 in the context of inflammatory disease. We are also preparing for an end of Phase 2 meeting with the FDA for HS-110 to discuss potential Phase 3 registration pathways."

"Additionally, we recently announced our new biosecurity/biodefense initiative, which leverages our gp96 platform against known or unknown future biological threats. In the short time since launching this program, we have filed patents to protect our proprietary technology and have established collaborations with leading institutions to advance our research. At the same time, we’ve assembled a knowledgeable and experienced biothreat advisory board that is well-versed on our biothreat platform. We plan to provide further updates on our biodefense efforts in the near future."

"Our strategic vision is to become a fully integrated, immune system-focused biopharmaceutical company, and we continue to evolve in that direction. In August we announced the groundbreaking of our new wholly-owned Scorpion biomanufacturing and bioanalytic subsidiary, which is designed to support our internal manufacturing needs as we advance our programs as well as to offer much-needed biomanufacturing/bioanalytic services to other biotech and pharmaceutical clients. Our ultimate goal is to reduce development costs, accelerate development timelines, and ensure the highest levels of quality by controlling the entire process internally. Overall, we have maintained a solid balance sheet and are well positioned to execute on a number of key milestones that we believe will drive significant value for shareholders."

Third Quarter 2021 Financial Results

Recognized $0.5 million of grant revenue for qualified expenditures under the CPRIT grant for the quarter ended September 30, 2021 compared to $0.8 million for the quarter ended September 30, 2020. The decrease in grant revenue in the current-year period primarily reflects the expected timing of completion of deliveries under the current phase of the contracts. As of September 30, 2021, we had a grant receivable balance of $0.9 million for CPRIT proceeds not yet received but for which the costs had been incurred or the conditions of the award had been met. We continue our efforts to secure future non-dilutive grant funding to subsidize ongoing research and development costs.
Research and development expense was $4.4 million and $3.2 million for the three months ended September 30, 2021 and 2020, respectively.
General and administrative expense was $3.4 million and $6.6 million for the three months ended September 30, 2021 and 2020. The decrease was primarily due to a decrease in stock-based compensation expense of $3.9 million.
Net loss attributable to Heat Biologics was approximately $7.4 million, or ($0.30) per basic and diluted share for the quarter ended September 30, 2021 compared to a net loss of approximately of $8.9 million, or ($0.43) per basic and diluted share for the quarter ended September 30, 2020.
As of September 30, 2021, the Company had approximately $108.9 million in cash, cash equivalents and short investments.

On November 10, 2021 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) (the "Company") reported that the company will deliver a corporate presentation during the 2021 Jefferies London Healthcare Conference — November 16-19, 2021 (Press release, Aurinia Pharmaceuticals, NOV 10, 2021, View Source [SID1234595089]). The presentation will be available to all conference attendees on demand (link here) beginning on Thursday, November 18 at 8 am GMT/3 am EST through Friday, November 19 at 5 pm GMT/12 pm EST.

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Interested parties can also register and access the on-demand webcast through the Investor section of the Aurinia corporate website – www.auriniapharma.com, under "News/Events."