On March 12, 2020 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing novel small molecule therapeutics to treat unmet needs in autoimmunity and cancer, reported its fourth quarter and year end 2019 financial results and corporate highlights (Press release, Kezar Life Sciences, MAR 12, 2020, View Source [SID1234555482]).

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"2019 marked several key milestones for Kezar, and I’m grateful for our team’s skill and hard work moving both of our programs forward. Our selective immunoproteasome inhibitor, KZR-616, entered Phase 2 trials in five different autoimmune diseases of high unmet need, building upon encouraging data from our ongoing Phase 1b study. We also nominated KZR-261, the first clinical candidate from our protein secretion program, and expect an IND filing in early 2021", said John Fowler, Chief Executive Officer. "This year we look forward to sharing updates from the Phase 1b portion of our MISSION study, as well as an interim analysis from our MARINA trial in AIHA and ITP. Finally, after our successful financing last month, we have a strong cash position that takes us beyond all of our KZR-616 Phase 2 readouts as well as data with KZR-261 in multiple tumor types."

Recent Clinical and Business Highlights

KZR-616 – Selective Immunoproteasome Inhibitor

MISSION Study

The Phase 1b/2 MISSION study in systemic lupus erythematosus (SLE) patients with and without nephritis is currently ongoing.

Additional data from the Phase 1b portion of MISSION will be presented during various medical conferences throughout the course of 2020.

The Phase 2 portion of MISSION is a randomized, placebo-controlled, double-blind trial to evaluate the safety and efficacy of KZR-616 in patients with active proliferative lupus nephritis. The primary endpoints of this portion of the MISSION trial are safety and tolerability. Secondary and exploratory endpoints include pharmacokinetics (PK), pharmacodynamics (PD), biomarker assessments and additional measures of efficacy. This trial includes four treatment arms evaluating KZR-616 administered subcutaneously once weekly for 24 weeks at dose levels of 30 mg, 45 mg and 60 mg, compared to placebo. The trial is designed to enroll up to 64 patients.

MARINA Study

The Phase 2 MARINA study in patients with autoimmune hemolytic anemia (AIHA) or immune thrombocytopenia (ITP) is currently ongoing.

MARINA is a Phase 2 randomized, dose-blind, multicenter trial designed to evaluate the safety, tolerability, efficacy, PK and PD of KZR-616 in patients with active AIHA or ITP. Patients with AIHA or ITP will be randomized to receive either 30 mg or 45 mg of KZR-616 subcutaneously once weekly for 13 weeks, followed by 12 weeks of follow-up. The trial is designed to enroll up to 40 patients. Kezar believes that whole blood RNASeq data from the Phase 1b portion of MISSION, which showed a decrease in multiple inflammatory gene signature and prolonged increase in erythropoietic gene signatures in SLE patients, support the broad potential anti-inflammatory activity of KZR-616 and therapeutic potential in patients with AIHA.

PRESIDIO Study

The Phase 2 PRESIDIO study in patients with polymyositis (PM) or dermatomyositis (DM) is currently ongoing.

PRESIDIO is a Phase 2 randomized, placebo-controlled, double-blind, crossover, multicenter trial to evaluate the safety, tolerability, efficacy, PK and PD of KZR-616 in patients with active PM or DM. During the 32-week treatment period, patients receive either 45 mg of KZR-616 or placebo subcutaneously once weekly for 16 weeks followed by a crossover to the other treatment arm for an additional 16 weeks. The trial is designed to enroll up to 24 patients. Kezar believes that KZR-616 has the potential to be developed into a treatment for patients with DM and PM, which is in-part supported by preclinical data in a mouse model of PM and DM that demonstrated immunoproteasome inhibition and improved muscle function.

KZR-261 – Protein Secretion Program

Research and discovery efforts targeting the protein secretion pathway have progressed significantly, and this novel program and pathway was featured in four separate presentations during two major medical and scientific conferences in 2019: the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) and the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting & Exposition (ASH) (Free ASH Whitepaper).

KZR-261, our first oncology clinical candidate and a first-in-class protein secretion inhibitor, has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies. Kezar anticipates filing an Investigational New Drug (IND) application for a Phase 1 study of KZR-261 in solid tumors in the first quarter of 2021.

Business Update

On February 4, 2020, Kezar completed an underwritten public offering of 18,965,385 shares of its common stock, which includes the full exercise of the underwriters’ option, and pre-funded warrants to purchase 2,884,615 shares of its common stock at an exercise price of $0.001 per share. The public offering price of the common stock was $2.60 per share and the public offering price of each pre-funded warrant was $2.599 per underlying share. The gross proceeds from the public offering were approximately $56.8 million, before deducting underwriting discounts and offering expenses.

Financial Results

Cash, cash equivalents and marketable securities totaled $78.2 million as of December 31, 2019, compared to $107.4 million as of December 31, 2018. The decrease in cash, cash equivalents and marketable securities was primarily attributable to cash used by the company in operations to advance its clinical stage programs as well as preclinical research and development.

Research and development expenses for the fourth quarter of 2019 increased by $2.7 million to $7.4 million compared to $4.7 million in the fourth quarter of 2018. Full year R&D expenses increased by $9.2 million in 2019 compared to 2018. This increase was primarily related to advancing the KZR-616 clinical program in multiple indications and the protein secretion preclinical program.

General and administrative expenses for the fourth quarter of 2019 increased by $0.8 million to $2.6 million compared to $1.8 million in the fourth quarter of 2018. Full year G&A expenses increased by $3.4 million in 2019 compared to 2018. The increase was primarily due to an increase in personnel expenses and costs related to operating as a public company.

Net loss for the fourth quarter of 2019 was $9.6 million, or $0.50 per basic and diluted common share, compared to a net loss of $5.8 million, or $0.30 per basic and diluted common share, for the fourth quarter of 2018. Net loss for 2019 was $35.1 million, or $1.84 per basic and diluted common share, compared to $23.2 million, or $2.26 per basic and diluted common share, in 2018.

Total shares outstanding were 19.2 million as of December 31, 2019. Additionally, there were outstanding options to purchase 3.1 million shares of common stock at a weighted average exercise price of $7.19 per share as of December 31, 2019.

About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Phase 1a clinical trial results in healthy volunteers provide evidence that KZR-616 potentially avoids adverse effects caused by currently marketed non-selective proteasome inhibitors, which Kezar believes prevent them from being utilized as a chronic treatment in autoimmune disorders. Phase 2 trials are underway for the treatment of lupus nephritis (MISSION study), autoimmune hemolytic anemia and immune thrombocytopenia (MARINA study), and dermatomyositis and polymyositis (PRESIDIO study).

About KZR-261

KZR-261, a novel, first-in-class protein secretion inhibitor, is the first clinical candidate to be nominated from Kezar’s research and discovery efforts targeting protein secretion pathways. KZR-261 is a broad-spectrum anti-tumor agent that acts through direct interaction and inhibition of Sec61 activity. The compound was discovered at Kezar through a robust medicinal chemistry campaign in which several

scaffolds were progressed through the company’s proprietary platform evaluating Sec61 modulation. As a result, Kezar has established a broad library of protein secretion inhibitors. KZR-261 has demonstrated several encouraging properties that lead to its potential to be an anti-cancer agent for the treatment of solid and hematologic malignancies. IND-enabling activities are currently underway, and an IND application in solid tumors is expected to be filed in the first quarter of 2021.

On March 12, 2020 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, reported financial and operating results for the fourth quarter and full year ended December 31, 2019 (Press release, Lineage Cell Therapeutics, MAR 12, 2020, View Source [SID1234555481]). Lineage management will host a conference call and webcast today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its fourth quarter and full year 2019 financial and operating results and to provide a business update.

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"2019 was a transformative year for Lineage. We established Lineage as a leading cell therapy company with the goal to usher in a new branch of medicine based on transplanting intact cells into the body to restore activity lost to aging, injury, or disease," stated Brian M. Culley, CEO. "We expanded our clinical pipeline by combining three complementary cell therapy assets under one roof, and also significantly reduced our cash burn by eliminating non-core activities. We focused our priorities and rebranded the company with a new leadership team, name, and headquarters in San Diego County. As we enter 2020, we intend to capitalize on the changes we made in 2019 and continue our positive momentum. Our primary goal is to complete enrollment in our Phase 1/2a clinical trial of OpRegen for dry AMD and collect the data that will guide our late-stage trial design and partnership discussions. We also will continue our efforts to advance the OPC1 program into a randomized trial by introducing commercially enabling enhancements to the manufacturing process. Lastly, we are working with our development partner, Cancer Research UK, and continue to assess clinical data that is being generated from the ongoing Phase 1 trial of VAC2. We are evaluating whether to exercise our option to acquire the data generated in the trial if the data supports that decision. We are excited about the three clinical programs that we are working to advance, each of which represents a potential billion dollar market opportunity."

Significant events and data updates from 2019 and early 2020 include:

Successfully dosed two patients in our OpRegen Phase 1/2a clinical trial for the treatment of dry age-related macular degeneration (AMD) with a new thaw-and-inject formulation and a new delivery device, the 510(k)-cleared Subretinal Delivery System (SDS) developed by Gyroscope Therapeutics (Gyroscope). The Gyroscope SDS is designed to precisely and consistently deliver therapeutics to the sub-retinal space via a suprachoroidal route, avoiding: (i) the need for a vitrectomy; (ii) perforation of the retina (retinotomy); and (iii) loss of cells and adverse safety events due to efflux. We have to date reported on two patients dosed with the combination of the new device and formulation. The first patient demonstrated notable improvements in vision, having gained 25 readable letters (or 5 lines) 6 months following administration, as assessed by the Early Treatment Diabetic Retinopathy Scale (ETDRS). This represented an improvement in visual acuity from a baseline of 20/250 to 20/100 in the treated eye. The second patient showed a small improvement in visual acuity in the treated eye at just 14 days following treatment. Both patients had rapid healing at the surgical site with no unexpected complications or any serious adverse events. Improvements for patients in the trial have typically become most evident approximately three to six months after treatment. Treatment for all patients in the trial continues to be well tolerated and all five patients in Cohort 4 (who have better baseline vision and less advanced disease than Cohorts 1-3 patients) registered improvement according to the ETDRS eye chart assessment. Improvements ranged from 8 to 25 additional letters correctly identified for all patients with at least 6 months of follow-up as of December 31, 2019. We expect to dose a total of six patients with the Gyroscope SDS under our current agreement with Gyroscope.
Acquired Asterias Biotherapeutics, Inc. in March 2019. As a result of this acquisition, we acquired two additional cell therapy product candidates, OPC1 and VAC2, along with their associated expansion opportunities and other assets. As we integrated the two companies, we reduced costs by about 50% by eliminating duplicate costs and rationalizing non-key projects.
Rebranded as Lineage Cell Therapeutics and relocated the corporate headquarters from the San Francisco Bay Area to Carlsbad, California. We also hired experienced biotech professionals to fill critical management positions, including the Chief Financial Officer, General Counsel and Vice President, Business Development.
Entered into agreements with three separate companies, with each agreement relating to different parts of Lineage’s intellectual property portfolio. All three companies have ongoing commercial operations in areas related to cell therapy. The aggregate up-front cash payment from the three transactions was greater than one million dollars with additional cash and royalties due upon reaching certain development milestones or product sales.
Reported a positive clinical update from our ongoing Phase 1/2a clinical trial of OPC1 known as SCiStar for the treatment of acute spinal cord injury (SCI). The overall safety profile of OPC1 remained excellent with robust motor recovery in the arms/hands maintained through year 2 follow-ups to date. Gains in motor function for patients assessed to date continued, representing tremendously meaningful improvements to quality of life and independence.
Awarded $3.2 million in grants from the Israel Innovation Authority and the National Institutes of Health and published or presented multiple papers and abstracts describing our work.
Obtained patents associated with the manufacture of our unique cell types, adding additional protections to all three of our clinical programs. Also obtained patent rights describing the use of induced pluripotent stem cells, an alternate option for generating differentiated cells for transplant and treatment of diseases, further broadening the potential application of our work.
Granted a Conformité Européenne (CE) Mark for Renevia, the Company’s facial aesthetics product, with an intended use in adults for the treatment of facial lipoatrophy. The CE Mark enables us to sell Renevia in Europe and we are actively pursuing a commercialization partner for this activity.
Announced that after reviewing promising preliminary data from the ongoing OpRegen Phase 1/2a clinical trial, our independent data safety monitoring board removed the protocol-mandated treatment stagger. Accordingly, we are opening two new U.S. clinical sites to accelerate patient enrollment and broaden surgical experience among dry AMD experts.
Potential key events for 2020:

Complete patient enrollment in the U.S. with the Gyroscope SDS in the ongoing Phase 1/2a clinical trial of OpRegen for the treatment of dry AMD.
Present new OpRegen data from the ongoing Phase 1/2a clinical trial in May 2020 and as available throughout the year.
Meet with the U.S. Food and Drug Administration (FDA) to discuss the further clinical development of OpRegen.
Evaluate partnership opportunities for the OpRegen program.
Enhance commercial utility of OPC1 program by introducing commercially enabling improvements to the manufacturing process in our GMP manufacturing facility.
Meet with the FDA to discuss the manufacturing and further clinical development of OPC1.
Provide further clinical updates from the SCiStar Trial for SCI.
Evaluate partnership opportunities for Renevia in Europe.
Evaluate VAC2 clinical data from the initial patients in the ongoing Phase 1 trial in NSCLC (non-small cell lung cancer) run by Cancer Research UK and evaluate potential early exercise of option to acquire data.
Continue engagement with the investment and medical communities with participation at medical and healthcare industry conferences, ongoing throughout 2020.
Strengthen existing partnerships with the National Institutes of Health, the Israel Innovation Authority, the California Institute for Regenerative Medicine, and Cancer Research UK.
Balance Sheet Highlights

Cash, cash equivalents, and marketable securities totaled $30.7 million as of December 31, 2019. Marketable securities include our remaining ownership of unrestricted securities in OncoCyte, AgeX Therapeutics, and Hadasit Bio-Holdings Ltd (Hadasit).

During 2019, we were able to fund our operations primarily by selling a portion of our marketable securities. We sold 6,250,000 shares of OncoCyte’s common stock for net proceeds of approximately $10.7 million. We also sold 765,889 shares of AgeX common stock for net proceeds of approximately $1.8 million and 1,048,147 shares of Hadasit common stock for net proceeds of approximately $1.7 million. On January 2, 2020, we sold 2,383,090 shares of OncoCyte stock for net proceeds of approximately $5.0 million. We continue to hold approximately 6 million shares of OncoCyte stock that are valued at $13.8 million as of March 10, 2020. All of our marketable securities are now in companies in which we hold less than 10% of the outstanding shares.

In conjunction with the sale of AgeX shares to Juvenescence Limited (Juvenescence) in 2018, we also hold a $21.6 million promissory note bearing 7% annual interest that matures on August 30, 2020. As of December 31, 2019, the outstanding principal and accrued interest on the note was $23.6 million. If, prior to August 30, 2020, Juvenescence completes an initial public offering resulting in gross proceeds of at least $50.0 million, the promissory note automatically converts into the Juvenescence securities. Lineage has the right to review Juvenescence’s financial statements twice per year.

In summary, as of December 31, 2019, the value of the Company’s cash, cash equivalents, marketable securities, and the balance of the Juvenescence promissory note due August 2020 were in excess of $54 million.

The Company has implemented significant cost savings initiatives and anticipates that net operational spend for 2020 will be approximately $16 million. This planned spending level represents a significant reduction from 2019 spending levels of $32 million and 2018 spending levels of $43 million for Lineage and Asterias combined. Lineage acquired Asterias on March 8, 2019. Assuming the Juvenescence note is paid in cash at maturity, the Company believes that it is funded well into 2021 as a result of these cost savings initiatives.

Fourth Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from royalties, licensing fees, research grants and the sale of research products. Total revenues for the three months ended December 31, 2019 were $1.2 million, an increase of $0.4 million as compared to $0.8 million for the same period in 2018. The increase was primarily related to a $0.7 million increase in royalties and licensing fees, offset by a $0.2 million decrease in grant revenue and $0.1 million decrease in the sale of research products.

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 December 31, 2019 were $8.0 million, a decrease of $2.8 million as compared to $10.8 million for the same period in 2018.

R&D Expenses: R&D expenses for the three months ended December 31, 2019 were $3.5 million, a decrease of $0.3 million as compared to $3.8 million for the same period in 2018. The decrease was primarily related to decreases of $0.7 million in Renevia and HyStem expenses and a decrease of $0.2 million in OpRegen expenses, offset by an increase of $0.6 million in OPC1 and VAC2 expenses (these programs were acquired in the Asterias merger).

G&A Expenses: G&A expenses for the three months ended December 31, 2019 were $4.5 million, a decrease of $2.5 million as compared to $7.0 million for the same period in 2018. The decrease was primarily attributable to a $1.8 million decrease in salaries, benefits and severance costs primarily related to terminated personnel, a $0.5 million reduction in severance, legal, accounting and other expenses related to the Asterias Merger, a $0.3 million reduction in accounting and consulting expenses and a $0.2 million reduction in travel expenses, offset by a $0.2 million increase in rent expense, which is primarily related to the implementation of ASC 842 Leases in 2019.

Loss from Operations: Loss from operations for the three months ended December 31, 2019 was $6.9 million, a decrease of $3.2 million as compared to $10.1 million for the same period in 2018.

Other Income/(Expenses), Net: Other income/(expenses), net for the three months ended December 31, 2019 reflected other income, net of $1.5 million, compared to other expense, net of ($35.2) million for the same period in 2018. The variance was primarily related to changes in the value of equity method investments and marketable equity securities for the applicable periods.

Net loss attributable to Lineage: The net loss attributable to Lineage for the three months ended December 31, 2019 was $4.5 million, or $0.03 per share (basic and diluted), compared to a net loss attributable to Lineage of $45.0 million, or $0.35 per share (basic and diluted), for the same period in 2018.

Full Year Operating Results

Note regarding AgeX: On August 30, 2018, Lineage deconsolidated AgeX from its consolidated financial statements due to the sale by Lineage of 14,400,000 shares of AgeX common stock to Juvenescence and the related decrease of Lineage’s ownership position in AgeX. Accordingly, Lineage ceased recognizing revenue and expenses related to AgeX and its programs on such date.

Revenues: Lineage’s revenue is generated primarily from royalties, licensing fees, research grants and the sale of research products. Total revenues for the year ended December 31, 2019 were $3.5 million, a decrease of $1.5 million as compared to $5.0 million for the same period in 2018. The decrease was primarily related to a $1.5 million decrease in grant revenue and a $0.7 million decrease in subscription and advertising revenues, partially offset by a $0.8 million increase in royalties from product sales and license fees.

Operating Expenses: Operating expenses are comprised of R&D expenses and G&A expenses. Total operating expenses for the year ended December 31, 2019 were $42.0 million, a decrease of $4.5 million as compared to $46.5 million for the same period in 2018.

R&D Expenses: R&D expenses for the year ended December 31, 2019 were $17.9 million, a decrease of $3.9 million as compared to $21.8 million for the same period in 2018. The decrease was primarily related to a decrease of $4.6 million related to the AgeX deconsolidation and the absence of AgeX R&D expenses incurred after August 30, 2018, a decrease of $3.8 million in Renevia and HyStem expenses and a decrease of $0.2 million in OpRegen expenses, offset by an increase of $4.8 million in OPC1 and VAC2 expenses (these programs were acquired in the Asterias merger).

G&A Expenses: G&A expenses for the year ended December 31, 2019 were $24.0 million, a decrease of $0.7 million as compared to $24.7 million for the same period in 2018. The decrease was primarily attributable to a $3.1 million decrease in AgeX related general and administrative expenses, a $1.4 million decrease in salaries, benefits and severance costs primarily related to terminated personnel, a $1.1 million reduction in legal and patent expenses and a $0.7 million reduction in consulting expenses, offset by a $5.6 million increase in severance, legal, accounting and other expenses related to the Asterias Merger.

Loss from Operations: Loss from operations for the year ended December 31, 2019 was $38.9 million, a decrease of $2.9 million as compared to $41.8 million for the same period in 2018.

Other Income/(Expenses), Net: Other income/(expenses), net for the year ended December 31, 2019 reflected other income, net of $19.6 million, compared to other expense, net of ($5.3) million for the same period in 2018. The variance was primarily related to the 2018 gain on the sale of AgeX shares and deconsolidation of AgeX and changes in the value of equity method investments and marketable equity securities for the applicable periods.

Net loss attributable to Lineage: The net loss attributable to Lineage for the year ended December 31, 2019 was $11.7 million, or $0.08 per share (basic and diluted), compared to a net loss attributable to Lineage of $46.0 million, or $0.36 per share (basic and diluted), for 2018.

Conference Call and Webcast

Lineage will host a conference call and webcast today, at 1:30 pm PT/4:30 pm ET to discuss its fourth quarter and full year 2019 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 March 20, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 3827019.

On March 12, 2020 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), an immunology company developing treatments that harness the patient’s innate immune system to fight disease, reported its financial results for the fourth quarter and full year ended December 31, 2019 and is providing a business update (Press release, INmune Bio, MAR 12, 2020, View Source [SID1234555480]).

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The Company will hold a conference call today at 11AM Eastern (U.S.) time. To participate in the live conference call, please dial 1-877-407-2988 five minutes prior to the scheduled conference call time and ask for the, "INmune Bio Conference Call." International callers should dial 1-201-389-0923. Participants can also use this link for instant telephone access to the event.

2019 and Recent Corporate Highlights on the DN-TNF Platform and NK Priming Platform:

·Closed an initial public offering ("IPO") and commenced trading on The Nasdaq Capital Market.
·Added two new independent members to the board of directors; Ed Baracchini former chief business officer of Xencor, Inc. who has negotiated many transactions with pharmaceutical and biotechnology companies and; Marcia Allen, a fortune 500 executive with vast accounting and finance experience.
DN-TNF Platform Highlights:

·Successfully reported Phase I clinical data that INB03 demonstrates safety and efficacy in cancer patients and has announced that the Phase II program will target trastuzumab resistance in women with HER2+ breast cancer using INB03 as part of combination therapy.
·Awarded a $1,000,000 grant from The Alzheimer’s Association allowing INMB to initiate a Phase 1 clinical trial using XPro1595 to treat patients with Alzheimer’s Disease.
·Awarded a $500,000 grant from The ALS Association to complete IND enabling pre-clinical studies with XPro1595 as a therapy for ALS.
·Initiated a program in NASH with LIVNate the third drug development program from the DN-TNF platform, joining DN-TNF platform drug candidates INB03 and XPro1595.

·XPro1595 had 9 articles published in peer review journals by academic collaborators and INB03 was one of 85 abstracts (out of 885) to be given as an oral presentation at SITC (Free SITC Whitepaper)2019.
·United States Patent and Trademark Office (USPTO) has issued Patent No. 15/776,061 on January 28, 2020, titled "CANCER PREVENTION AND THERAPY BY INHIBITING SOLUBLE TUMOR NUCROSIS FACTOR", which covers INmune’s INB03 Program utilizing dominant negative TNF (DN-TNF) technology for treating cancer.
NK Priming Platform Highlights:

·Announced publication of data on INKmune in Peer-Reviewed Journal PLOS ONE. INKmune is a novel NK cell priming platform that signals the patient’s own NK cells to target residual disease of cancer.
·Finalized and validated manufacturing and distributions processes for INKmune to support up-coming clinical trials.
Professor Lowdell, CSO INmune Bio and Professor of Cell Therapy at UCL, London, and creator of the INKmune product said, "2019 has been a landmark year for this product and is the culmination of 20 years’ of bench-to-bedside research. We have published the molecular basis of the mechanism of action in a peer-reviewed journal, developed scaled manufacture adequate for full commercial delivery and validated an efficient and easily delivered cold supply chain for the drug. I am extremely excited about our delivery of patients into two phase I trials this year in high risk MDS cancer and ovarian cancer, two very different clinical applications."

"2019 and year to date, has been a period of substantial progress," stated RJ Tesi, M.D., Chief Executive Officer of INmune Bio. "We completed our first-in-man study of our DN-TNF platform in cancer and have expanded that franchise to include Alzheimer’s Disease and NASH. Additionally, we just announced pre-clinical proof-of-concept studies in ALS. Both our NK cell priming platform and DN-TNF platform continue to generate data in the laboratory and the clinic to allow us to expand the depth and breadth of our pipeline."

Upcoming Milestones:

·Report results of Phase 1 XPro1595 in Alzheimer’s Disease, expected to complete 2H 2020.
·Enroll first patient in Phase II INB03 program, targeting trastuzumab resistant HER2+ breast cancer using INB03 as part of combination therapy, expected mid-2020.
·Enroll first patient in Phase II LIVNate for NASH, expected mid-2020.
·Enroll first patient in Phase I INKmune in High Risk MDS cancer, expected 2H 2020.
·Enroll first patient in Phase I INKmune in Ovarian cancer, expected 2H 2020.

Financial Results for the Fourth Quarter Ended December 31, 2019:

Net loss attributable to common stockholders for the fourth quarter ended December 31, 2019 was $2.3 million, compared to $1.9 million for the quarter ended December 31, 2018.

Research and development expense totaled approximately $0.9 million for the fourth quarter ended December 31, 2019, compared with approximately $1.0 million for the quarter ended December 31, 2018. The decrease in research and development expense was due to the Company recording $0.3 million of contra research and development expense as a result of the receipt of a grant from the Alzheimer’s Association. Excluding the grant, research and development expense increased during the three months ended December 31, 2019 as a result of the further advancement of our drug platforms.

General and administrative expense was approximately $1.5 million in the quarter ended December 31, 2019, compared to approximately $0.9 million in the quarter ended December 31, 2018. The $0.6 million increase in general and administrative expense is largely due to the costs associated with being a public company and due to higher compensation cost (including higher stock-based compensation).

At December 31, 2019, the Company had cash and cash equivalents of approximately $7.0 million with no debt. During the quarter ending December 31, 2019, the Company received $0.4 million of cash proceeds from Australia and $0.4 million of cash proceeds from the United Kingdom pursuant to research and development tax credits.

As of March 10, 2020, the Company had 10.7 million common shares outstanding.

On March 12, 2020 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the fourth quarter and fiscal year ended December 31, 2019 (Press release, CymaBay Therapeutics, MAR 12, 2020, View Source [SID1234555479]).

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"Since announcing the decision to halt the development of seladelpar last November, we have been focused on two parallel initiatives: an investigation of the unexpected histologic findings identified by study pathologists in the Phase 2b study of seladelpar in NASH, and an evaluation of strategic alternatives to maximize shareholder value," said Sujal Shah, President and CEO of CymaBay. "Our investigation includes several key activities that will be essential for us to understand the nature and significance of the findings and have the requisite follow-up dialogue with the FDA which we are planning for before the end of the second quarter."

Shah continued, "In parallel to this investigation, our executive team and board have been focused on a comprehensive evaluation of strategic alternatives and cost-cutting initiatives. While we remain committed to completing the investigation, we believe these efforts are prudent in order to make decisions expeditiously once we gain needed clarity on the potential path forward for seladelpar."

Recent Business Highlights

The development of seladelpar was halted in all indications after consulting with expert liver pathologists and hepatologists and in consideration of patient safety. The FDA agreed with this decision, formally placed the seladelpar program on clinical hold for all indications, and subsequently provided input on plans to further investigate the situation. Since then, CymaBay has commenced an in-depth review of the Phase 2b NASH findings. This investigation includes three activities intended to confirm and subsequently understand the significance of the findings identified by study pathologists:
First, a comprehensive collection and review of data including patient demographics, medical history, concomitant medications and a broad set of biochemical markers.
Second, a blinded, independent review of baseline and end of treatment biopsies by several, world-renowned liver pathologists. The independent pathology review will include an accepted pathology scoring framework, known as the Ishak Modified HAI scoring system, to quantitatively characterize features of histology present in our patient population both at baseline and at end-of-treatment. Among these features includes a scoring for the presence and severity of interface hepatitis which is not quantified in the existing framework for scoring NASH pathology.
Third, a formal pathology and clinical hepatology review panel meeting that CymaBay anticipates convening in the middle of the second quarter during which experts will review all information gathered to provide a consensus and independent determination of the role of seladelpar in the Phase 2b NASH findings. This panel will allow for a properly informed dialogue with FDA regarding seladelpar development.

Completed reading of the Phase 2b NASH end-of-treatment biopsies by study pathologists. Preliminary results reported below are for the 152 patients out of the 181 patients enrolled in the study with paired biopsies at entry and end-of-study:
Phase 2b Preliminary Topline Results

Resolution of NASH defined as patients having a NAFLD Activity Score (NAS) of 0 or 1 for lobular inflammation and 0 for hepatocellular ballooning.
Patients that did not have end-of-study biopsies are not included in reported histology endpoints.
Measured changes from baseline to end-of-treatment in liver enzymes including ALT, AST, GGT and ALP, resembled the pattern of meaningful reductions previously reported at week 12.
Implemented cost containment and restructuring program following the decision to place the seladelpar program on hold in order to minimize expenses and conserve capital. As part of this program, CymaBay froze hiring, significantly scaled-back future procurement plans, reduced its work force by more than 60% and scaled down or cancelled many existing contracts for goods and services. The size of the Board of Directors has also been decreased from nine to five seats.

As a result of these actions, CymaBay recorded a $5.1 million restructuring charge during the fourth quarter which includes $2.9 million of employee severance costs, $0.9 million of non-cash stock-based compensation expense associated with the acceleration of stock options of certain terminated employees, and $1.3 million of charges associated with the termination of certain contract manufacturing agreements.

Held $190.9 million in cash, cash equivalents and short-term investments at December 31, 2019.
Fourth Quarter and Year Ended December 31, 2019 Financial Results

Research and development expenses for the three and twelve months ended December 31, 2019 were $20.9 million and $83.8 million, respectively. This compared to R&D expenses of $16.4 million and $58.1 million for the three and twelve months ended December 31, 2018, respectively. Prior to the decision to halt development of seladelpar in November 2019, research and development expenses in the fourth quarter and twelve months ended 2019 were generally higher than in the corresponding periods in 2018 due to expanding clinical trial activities related to the PBC Phase 3 clinical trial, PSC Phase 2 clinical trial, and other NDA-enabling studies.

General and administrative expenses for the three and twelve months ended December 31, 2019 were $4.5 million and $19.2 million, respectively. This compared to $4.2 million and $14.4 million for the three and twelve months ended December 31, 2018, respectively. Prior to the decision to halt development of seladelpar, G&A expenses in the fourth quarter and twelve months ended 2019 were higher than in the corresponding periods in 2018 as a result of higher labor costs and other administrative expenses necessary to support expanding development activities.

Net loss for the three and twelve months ended December 31, 2019 was $29.4 million, or ($0.43) per diluted share, and $102.8 million, or ($1.53) per diluted share, respectively. This compared to net loss of $19.4 million, or ($0.32) per diluted share, and $72.5 million, or ($1.26) per diluted share, in the three and twelve months ended December 31, 2018, respectively. Net loss was higher largely due to increases in operating expenses, including restructuring charges.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss fourth quarter and fiscal year end 2019 financial results and provide a business update. To access the live conference call, please dial 855-327-6837 from the U.S. and Canada, or 631-891-4304 internationally, Conference ID# 10008868. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On March 12, 2020 Tyligand Bioscience, Ltd., a leader in small molecule drug discovery and development, and Context Therapeutics LLC, a clinical stage biopharmaceutical company focused on hormone driven cancers, reported the signing of collaboration agreements for the manufacturing, registration and future commercialization of onapristone extended release (ER) (Press release, Context Therapeutics, MAR 12, 2020, View Source [SID1234555478]).

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Onapristone ER is currently being evaluated in patients with progesterone receptor positive (PR+) rare ovarian and endometrial cancers in the ongoing Phase 2 ONWARD 220 clinical trial. Initiation of additional Phase 2 clinical trials in ER+, PR+, HER2- breast cancer and PR+ endometrial cancers are planned for 2020.

Under the terms of the agreements, Tyligand will be solely responsible for the design and optimization of a novel manufacturing process for onapristone ER to meet Context’s development and future commercialization needs, and standards for quality, safety and cost. Upon completion of specific performance-based milestones, Tyligand will be granted the exclusive right and will be solely responsible for the development and commercialization of onapristone ER in China, Hong Kong and Macau (the "Territory"), and Context will be eligible to receive royalties on net sales of onapristone ER in the Territory. Context will retain rest of world rights to commercialize onapristone ER.

"We are thrilled to partner with Tyligand as we accelerate onapristone ER’s Phase 2 evaluation and prepare for Phase 3," said Martin Lehr, CEO of Context. "Tyligand is renowned for its expertise in process development and has strong networks with manufacturing and clinical capabilities in China and the U.S. Partnering with Tyligand will enable Context to optimize and efficiently scale our manufacturing and clinical capacity to support the evaluation and future commercialization of onapristone ER, our experimental oral therapy, to address the unmet need in treating patients with PR+ cancers."

"Even with the major advances in cancer therapies in recent years, treatment options for patients with hormone driven cancers remain limited," said Tony Zhang, CEO of Tyligand. "Onapristone ER has the potential to be the first-in-class therapeutic agent specifically targeting progesterone receptors and the best-in-class treatment option for breast, endometrial and ovarian cancers. We are proud to partner with Context to develop onapristone ER and make this innovative medicine ultimately more accessible for patients around the world."

About Onapristone ER
Onapristone ER (extended release) is a potent and specific antagonist of the progesterone receptor that is orally administered. Currently, there are no approved therapies that selectively target progesterone receptor positive (PR+) cancers. Preclinical and clinical data suggest that onapristone ER has anticancer activity by inhibiting progesterone receptor binding to chromatin, downregulating cancer stem cell mobilization and blocking immune evasion. Onapristone ER is currently being evaluated in patients with PR+ rare ovarian and endometrial cancers in the ongoing Phase 2 ONWARD 220 clinical trial. Additional Phase 2 clinical trials in ER+, PR+, HER2- breast cancer and PR+ endometrial cancers will be initiated in 2020. Onapristone ER is an investigational drug that has not been approved for marketing by any regulatory authority.