On August 6, 2020 Bellicum Pharmaceuticals, Inc. (NASDAQ: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the second quarter 2020 and provided an operational update (Press release, Bellicum Pharmaceuticals, AUG 6, 2020, View Source [SID1234563091]).

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"Bellicum continued to make significant progress in the second quarter, highlighted by FDA clearance of our IND for BPX-603, our first dual-switch Go-CAR-T product candidate to enter clinical development," said Rick Fair, President and Chief Executive Officer of Bellicum. "We expect to be entering a period of accelerated news flow for our company with multiple data readouts planned in the next 24 months, starting in the fourth quarter with our initial presentation of the BPX-601 Phase 1 cohort 5C with repeat rimiducid."
Program Highlights and Current Updates

BPX-601 GoCAR-T
•Bellicum continues to enroll patients in cohort 5C of the BPX-601 clinical trial in second-line metastatic pancreatic cancer to evaluate the safety of repeat rimiducid dosing to re-activate BPX-601 cells over time. Bellicum plans to present interim results for this cohort at a medical meeting by the end of the year. The study has experienced modest enrollment delays and data collection issues related to the COVID-19 pandemic which may impact the number of patients, duration of follow-up and data to be presented.

•In addition, Bellicum has submitted an amendment to the FDA to modify the BPX-601 trial by: (1) expanding eligibility to third-line pancreatic cancer patients; (2) extending dose escalation of BPX-601 cells to 1×107 cells/kg; and (3) opening a cohort in relapsed metastatic castration-resistant prostate cancer. The company expects to begin enrolling under this amended protocol later this year.

BPX-603 GoCAR-T
•In June 2020, Bellicum received clearance from the U.S. Food and Drug Administration (FDA) of its investigational new drug application (IND) for BPX-603. BPX-603 is a GoCAR-T product candidate targeting solid tumors that express human epidermal growth factor receptor 2 (HER2). BPX-603 is the company’s first dual-switch GoCAR-T product candidate, which incorporates both the company’s iMC activation and CaspaCIDe safety switch technologies and the company plans to initiate a Phase 1/2 basket trial in solid tumors that express HER2 later this year.

BCMA GoCAR-NK Program
•In May 2020, preclinical data from Bellicum’s GoCAR-NK cell program was published in the digital edition of Blood Advances, a journal published by The American Society of Hematology (ASH) (Free ASH Whitepaper), illustrating that the incorporation of iMC into CAR-NK cells was found to improve cell proliferation and persistence, stimulate cytokine production, and enhance innate cytotoxicity against tumor cells in multiple models. Preclinical development activities are continuing for Bellicum’s GoCAR-NK program targeting B-cell maturation antigen, or BCMA, for the treatment of multiple myeloma. Management expects to present additional preclinical data for this program by the end of 2020.

Corporate Highlights

•In April 2020, Bellicum closed a transaction in which The University of Texas MD Anderson Cancer Center acquired Bellicum’s approximately 60,000-square-foot Houston facility, including manufacturing, office and laboratory space, for $15.0 million. Concurrent with this transaction, Bellicum partially repaid approximately $7.0 million of its Oxford Finance debt obligations.

Second Quarter 2020 Financial Results
R&D Expenses: Research and development (R&D) expenses were $11.8 million and $22.2 million for the three and six months ended June 30, 2020, respectively, compared to $20.0 million and $36.9 million for the three and six months ended June 30, 2019, respectively. The reduction in expenses in the second quarter and first half of 2020 resulted primarily from reduced expenses related to reduced rivo-cel related activities, reduced expenses resulting from the manufacturing facility sale and the reduction in force implemented during the second half of 2019, partially offset by an increase in expenses related to the GoCARTM programs.

G&A Expenses: General and administrative (G&A) expenses were $3.8 million and $7.9 million for the three and six months ended June 30, 2020, respectively, compared to $7.5 million and $15.1 million for the three and six months ended June 30, 2019, respectively. The reduction in expenses during the second quarter and first half of 2020 relative to the comparable period in 2019 was primarily due to the reduction in rivo-cel related commercialization activities as well as the effects of the reduction in force that reduced employee-related charges.

Loss from Operations: Bellicum reported a loss from operations of $11.8 million and $26.4 million for the three and six months ended June 30, 2020, respectively, compared to a loss from operations of $26.2 million and $50.0 million for the three and six months ended June 30, 2019, respectively. The results for the three and six months ended June 30, 2020 included a net gain on dispositions of $3.8 million due to the manufacturing facility sale. Cash used in operating activities was $30.5 million for the six months ended June 30, 2020, compared to cash used in operating activities of $46.0 million for the six months ended June 30, 2019.

Net Loss: Bellicum reported net loss of $43.2 million and $25.6 million for the three and six months ended June 30, 2020, respectively, compared to a net loss of $26.9 million and $51.5 million for the three and six months ended June 30, 2019, respectively. The results included a non-cash loss of $30.7 million and non-cash gain of $2.1 million related to the change in fair value of warrant and private placement option liability for the three and six months ended June 30, 2020, respectively.
Shares Outstanding: As of June 30, 2020, Bellicum had 5,059,779 shares of common stock and 534,000 shares of preferred stock outstanding. Each share of preferred stock can be converted into 10 shares of common stock.
Cash Position and Guidance: Bellicum reported cash and cash equivalents and restricted cash totaling $68.0 million as of June 30, 2020, compared to $93.8 million as of December 31, 2019. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into the second half of 2021. Management expects cash utilization of $55 to $65 million in 2020.

Potential Milestones

Below is a summary of notable anticipated milestones for our ongoing programs:

BPX-601
•Initial Phase 1 cohort 5C data (repeat rimiducid) – pancreatic cancer: Q4’20
•Phase 1/2 data update – pancreatic cancer: 2H’21
•Initial Phase 1/2 data – prostate cancer: 2022

BPX-603
•Initiate Phase 1/2 trial: 2H’20
•Initial Phase 1 data: 2H’21
•Phase 1 data update: 2022

BCMA GoCAR-NK
•Preclinical data update: Q4’20
•Preclinical data update: 2H’21
•IND clearance: 2022

Conference Call and Webcast

Bellicum’s management will host a webcast and conference call today at 5 p.m. ET / 2 p.m. PT, August 6, 2020, to discuss the financial results for the second quarter 2020 and provide a corporate update. The live call may be accessed by dialing (800) 920-0677 for domestic callers and (212) 231-2908 for international callers. A live webcast of the call will be available from the Investors and Media section of the company’s website at www.bellicum.com and a replay will be available shortly after the live event.

On August 6, 2020 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, reported recent business highlights, anticipated upcoming milestones, and financial results for the second quarter and first half of 2020 (Press release, BeiGene, AUG 6, 2020, View Source [SID1234563090]).

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"We have made tremendous progress since the start of the second quarter, with approvals for tislelizumab and BRUKINSA for three indications in China and eight accepted new drug applications for tislelizumab, BRUKINSA, and pamiparib in China, the European Union, Australia, and Israel. Our commercial teams grew product sales to a new quarterly high of approximately $66 million, driven by our recently launched internally developed products," said John V. Oyler, Co-Founder, Chief Executive Officer, and Chairman of BeiGene. "We recently completed a very successful registered direct offering in which we raised net proceeds of approximately $2.07 billion and believe that we are well-positioned to accelerate the development of our deep pipeline, further expand our portfolio in oncology and into other therapeutic areas, and continue to build our capabilities and operations for our products to serve more patients worldwide. In the remainder of 2020 and 2021, we look forward to key clinical readouts, as well as expanded commercial opportunities for our products through approvals in additional indications and geographic markets and by growing our commercial-stage portfolio to up to 11 products."
Recent Business Highlights and Upcoming Milestones

Commercial Operations
•Received approval from the National Medical Products Administration (NMPA) for BRUKINSA (zanubrutinib) in China in June for the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least one prior therapy, and for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. Subsequently launched BRUKINSA in these indications in China within 12 days of approval;
•Received approval from the NMPA and launched tislelizumab in China in April for the treatment of patients with previously treated locally advanced or metastatic urothelial carcinoma (bladder cancer);
•Began commercializing XGEVA (denosumab) in China on July 1 for the treatment of giant cell tumor of bone (GCTB), following its earlier approval by the NMPA in May 2019 and subsequent launch by Amgen. This marks the first Amgen product that has been transitioned to BeiGene for commercialization in China since the commencement of our global strategic oncology collaboration in January 2020;
•Generated $65.64 million in product revenue in the three months ended June 30, 2020, representing a 13% increase compared to the comparable period of the prior year, despite the suspension and recall of ABRAXANE in China in March 2020. Product revenue was driven by sales of our newly launched internally developed products tislelizumab and BRUKINSA; and
•Received supplemental medical insurance coverage in Hainan province, China for tislelizumab for patients with classical Hodgkin’s lymphoma (cHL), BRUKINSA for patients with CLL/SLL, and XGEVA for patients with GCTB.
Development Programs
BRUKINSA (zanubrutinib), a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to maximize BTK occupancy and minimize off-target effects. BRUKINSA has received accelerated approval in the United States for the treatment of adult patients with MCL who have received at least one prior therapy; and in China in two indications – the treatment of adult patients with CLL/SLL who have received at least one prior therapy, and the treatment of adult patients with MCL who have received at least one prior therapy. BRUKINSA is under development globally for additional approvals.
•Announced the acceptance of a marketing authorization application (MAA) by the European Medicines Agency (EMA) for BRUKINSA for the treatment of patients with Waldenström’s Macroglobulinemia (WM) who have received at least one prior therapy or as first-line treatment for patients unsuitable for chemo-immunotherapy;
•Presented clinical data from the Phase 3 ASPEN trial comparing BRUKINSA to ibrutinib for the treatment of patients with WM at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program;
•Announced an exclusive distribution agreement with Medison Pharma Ltd. in Israel and the acceptance of a new drug application (NDA) in Israel for BRUKINSA for the treatment of patients with MCL who have received at least one prior therapy; and
•Filed and received acceptance of two NDAs by the Australia Therapeutic Goods Administration (TGA) for BRUKINSA in relapsed/refractory (R/R) MCL and WM.
Expected Milestones for BRUKINSA
•File a supplemental new drug application (sNDA) in China for WM in 2020;
•Announce top-line results from the SEQUOIA trial (NCT03336333) comparing BRUKINSA with bendamustine plus rituximab in patients with treatment-naïve CLL or SLL as early as the second half of 2020;
•Discuss data from the Phase 3 ASPEN trial (NCT03053440) comparing BRUKINSA to ibrutinib in patients with WM with the U.S. Food and Drug Administration (FDA) in 2020; and
•Complete expanded enrollment in the Phase 3 ALPINE trial (NCT03734016) comparing BRUKINSA with ibrutinib in patients with R/R CLL/SLL in 2020.
Tislelizumab, a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages; approved in China in two indications – the treatment for patients with cHL who received at least two prior therapies, and the treatment of patients with locally advanced or metastatic urothelial carcinoma with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. Tislelizumab is under development globally for additional approvals.
•Announced that the NMPA accepted a sNDA for tislelizumab in combination with chemotherapy for first-line treatment of patients with advanced non-squamous non-small cell lung cancer (NSCLC);
•Announced that the NMPA accepted a sNDA for tislelizumab for the treatment of patients with previously treated unresectable hepatocellular carcinoma (HCC), the most common form of liver cancer;
•Presented results from a Phase 3 clinical trial evaluating tislelizumab in combination with standard chemotherapy for the first-line treatment of patients with advanced squamous NSCLC at the 2020 ASCO (Free ASCO Whitepaper) Virtual Scientific Program. Data from this trial were included in the sNDA currently under review by the NMPA;
•Initiated patient enrollment in a Phase 3 trial (NCT04379635) in China comparing tislelizumab plus chemotherapy to placebo plus chemotherapy in patients with resectable Stage II or IIIA NSCLC;
•Completed enrollment in the pivotal Phase 2 trial (NCT03736889) in China of patients with mismatched repair deficient (dMMR) or microsatellite instability-high (MSI-H) solid tumors; and
•Entered into a clinical collaboration agreement with Hutchison China MediTech Limited (Chi-Med) to evaluate the safety, tolerability and efficacy of combining tislelizumab with two of Chi-Med’s drug candidates, ELUNATE (fruquintinib) and surufatinib, for the treatment of various solid tumor cancers in the United States, Europe, China, and Australia.
Expected Milestones for Tislelizumab
•Present data from the Phase 3 trial (NCT03663205) of tislelizumab combined with chemotherapy for the first-line treatment of patients with advanced non-squamous NSCLC at the 2020 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress. Data from this trial were included in the sNDA currently under review by the NMPA; and
•Announce top-line results from the global Phase 3 trial (NCT03358875) comparing tislelizumab versus docetaxel in second-or third-line patients with NSCLC and the global Phase 3 trial (NCT03430843) comparing tislelizumab versus chemotherapy in second-line patients with advanced esophageal squamous cell carcinoma (ESCC) in 2020 or early 2021.
Pamiparib, an investigational selective small molecule inhibitor of PARP1 and PARP2
•Announced that the NMPA accepted and subsequently granted priority review of an NDA for pamiparib for the treatment of patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy.
Expected Milestones for Pamiparib
•Present data from the Phase 1/2 trial (NCT03333915) of pamiparib in patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy, at the 2020 ESMO (Free ESMO Whitepaper) Virtual Congress. Data from this trial were included in the NDA currently under review by the NMPA; and
•Announce top-line results from the Phase 3 trial (NCT03519230) of pamiparib as a maintenance treatment in patients with platinum-sensitive recurrent ovarian cancer (OC) in 2020 or the first half of 2021.
Early-Stage Clinical Development Programs
•Continued to advance our earlier-stage pipeline of internally-developed assets, including BGB-A1217 (monoclonal antibody against TIGIT in Phase 1/2 development for cancer in combination with tislelizumab), BGB-11417 (BCL-2 inhibitor in Phase 1 development for cancer), BGB-A445 (non-ligand competing OX40 monoclonal antibody in Phase 1 development for solid tumors in combination with tislelizumab), BGB-10188 (PI3Kδ inhibitor in Phase 1 development for cancer in combination with BRUKINSA or tislelizumab), and BGB-15025 (HPK1 inhibitor in preclinical development for cancer); and
•Identified a recommended Phase 2 dose in the Phase 1 trial (NCT04047862) of BGB-A1217 (TIGIT monoclonal antibody) in combination with tislelizumab in advanced solid tumors.
Collaboration Programs
Zanidatamab (ZW25), a novel investigational Azymetric bispecific antibody against HER2 currently in Phase 2 clinical development with Zymeworks Inc.
•Began the manufacturing technology transfer to our biologics facility in Guangzhou.
Expected Milestones for Zanidatamab
•Support clinical development and enrollment of the planned registration-enabling trials in refractory HER2-positive biliary tract cancer in 2020 and first-line HER2-positive gastroesophageal adenocarcinomas in late 2020 or early 2021.
BGB-3245, an investigational RAF dimer inhibitor with activity against mutant monomeric and dimeric forms of B-RAF in preclinical studies; being developed by MapKure, which is jointly owned by BeiGene and SpringWorks Therapeutics
•Initiated patient enrollment in the United States and Australia in the Phase 1 clinical trial (NCT04249843) of BGB-3245 in patients with advanced or refractory solid tumors.
Expected Milestones for BGB-3245
•Initial clinical data from the Phase 1 study expected in 2021.
Manufacturing Facilities
•Completed equipment validation and continued manufacturing process validation for the first phase of our biologics manufacturing facility in Guangzhou; and
•Initiated expansion of the second phase of our biologics manufacturing facility in Guangzhou to significantly increase manufacturing capacity and introduce new manufacturing technology platforms, expected to be completed by the end of 2020.
COVID-19 Impact and Response
•The Company expects that the worldwide health crisis of COVID-19 will continue to have a negative impact on its operations, including commercial sales, regulatory interactions and inspections, and clinical trial recruitment and participation. Although the impact of COVID-19 on operations in China lessened in the second quarter of 2020 compared to the first quarter of 2020, there remains uncertainty regarding the future impact of the pandemic both in China as well as globally. The Company is striving to minimize delays and disruptions, and continues to execute on its commercialization, regulatory and clinical development goals globally.
Other Developments
•Announced the closing of a registered direct offering of 145,838,979 ordinary shares, at a price of $14.2308 per share ($185 per American Depositary Share (ADS)), resulting in gross proceeds of approximately $2.08 billion and net proceeds, after estimated offering expenses, of approximately $2.07 billion.
•Announced a license and collaboration agreement with Assembly Biosciences, Inc. in China for its portfolio of three clinical-stage chronic hepatitis B virus (HBV) core inhibitor candidates ABI-H0731, ABI-H2158, and ABI-H3733, for the treatment of patients with HBV infection; and
•Announced the appointment of Angus Grant, Ph.D. as Chief Business Executive. Dr. Grant will oversee business development and alliance management and help drive external innovation and investment to guide our global growth strategy.
Second Quarter 2020 Financial Results
Cash, Cash Equivalents, Restricted Cash, and Short-Term Investments were $3.16 billion as of June 30, 2020, compared to $3.38 billion as of March 31, 2020, and $985.50 million as of December 31, 2019. Our cash balance as of June 30, 2020 does not include net proceeds of approximately $2.07 billion received on July 15, 2020 from a registered direct offering of our ordinary shares to certain existing shareholders.
•In the three months ended June 30, 2020, cash used in operating activities totaled $263.00 million and capital expenditures were $32.61 million, compared to $46.10 million and $21.45 million, respectively, in the prior year period.
Revenue for the three months ended June 30, 2020 was $65.64 million, compared to $243.35 million in the same period of 2019. The decrease in total revenue is primarily attributable to the absence of collaboration revenue after the termination of the Celgene collaboration for tislelizumab in June 2019 and decreased product sales of ABRAXANE in China following the suspension by the NMPA and recall in March 2020, partially offset by sales of tislelizumab in China and BRUKINSA in the United States and China.
•Product revenues totaled $65.64 million for the three months ended June 30, 2020, compared to $58.14 million for the same period in 2019, comprised of:
–$29.42 million from sales of tislelizumab in China, in its first full quarter of sales following its launch in March 2020;
–$6.97 million from sales of BRUKINSA in China and the United States, including the launch inventory build at distributors following approval in China in June 2020;
–$29.01 million from sales of REVLIMID and VIDAZA in China, compared to $23.41 million in the same period of the prior year; and
–$0.24 million from ABRAXANE, which was comprised of reversals of rebate accruals subsequent to the suspension of sales and recall of ABRAXANE in March 2020, compared to $34.73 million in the same period of the prior year.
•Collaboration revenue was nil for the three months ended June 30, 2020, compared to $185.20 million for the same period in 2019. Collaboration revenue for the three months ended June 30, 2019 included a termination fee of $150 million from the termination of the Celgene collaboration agreement for tislelizumab, as well as $25.74 million of research and development service revenue and $9.46 million of reimbursed research and development costs under the agreement, both of which were recognized prior to termination of the agreement.
Expenses for the three months ended June 30, 2020 were $424.51 million, compared to $329.18 million in the same period of 2019.
•Cost of Sales for the three months ended June 30, 2020 were $14.31 million, compared to $17.84 million in the same period of 2019. Cost of sales primarily included acquisition costs for supply of REVLIMID and VIDAZA that was sold during the period in China, as well as the post-approval costs of tislelizumab and BRUKINSA that was sold during the period.
•R&D Expenses for the three months ended June 30, 2020 were $285.97 million, compared to $228.76 million in the same period of 2019. The increase in R&D expenses was primarily attributable to continued increases in spending on
our ongoing and newly initiated late-stage pivotal clinical trials, development expenses associated with the Amgen collaboration, the preparation for additional regulatory submissions, and manufacturing costs related to pre-commercial activities and supply. Our co-funding obligation for the development of the pipeline assets under the Amgen collaboration for the three months ended June 30, 2020 was $55.94 million, of which $28.34 million was recorded as R&D expense. The remaining $27.61 million was recorded as a reduction of the R&D cost share liability. R&D-related share-based compensation expense was $23.71 million for the three months ended June 30, 2020, compared to $18.15 million for the same period of 2019.
•SG&A Expenses for the three months ended June 30, 2020 were $124.05 million, compared to $82.25 million in the same period in 2019. The increase in SG&A expenses was primarily attributable to increased headcount, including the expansion of our commercial team to support the distribution of our products in China and the United States, as well as higher professional service fees and costs to support our growing operations. SG&A-related share-based compensation expense was $21.76 million for the three months ended June 30, 2020, compared to $14.45 million for the same period of 2019.
•Net Loss for the three months ended June 30, 2020 was $335.20 million, or $0.33 per share, or $4.31 per ADS, compared to $85.57 million, or $0.11 per share, or $1.43 per ADS in the same period in 2019.

On August 6, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported its financial results for the second quarter and six months ended June 30, 2020 (Press release, CTI BioPharma, AUG 6, 2020, View Source [SID1234563089]).

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"This past quarter we announced enrollment of the first patient in our PRE-VENT Phase 3 clinical trial of pacritinib in hospitalized patients with severe COVID-19, an important step for CTI as we work to provide a new therapeutic option for COVID-19 patients," said Adam R. Craig, M.D., Ph.D. "With regards to the PACIFICA Phase 3 trial, we continue to enroll patients but the enrollment rate is lower than planned due to the COVID-19 pandemic and we now anticipate at least a six-month delay in the trial. However, given our cash runway into Q4 2021, we remain confident in our ability to successfully execute on the development of pacritinib for the treatment of severely thrombocytopenic myelofibrosis patients."

Second Quarter Financial Results
Operating loss was $10.0 million and $21.9 million for the three and six months ended June 30, 2020, respectively, compared to operating loss of $11.0 million and $21.5 million for the respective periods in 2019. Operating loss for the three months ended June 30, 2020 as compared to the comparable period in 2019 resulted primarily from a decrease in general and administrative expenses. The increase in operating loss for the six months ended June 30, 2020 as compared to the comparable period in 2019 resulted primarily from the recording of a full allowance against certain VAT receivables due to an increased uncertainty of collectability.

No revenues were recognized for the three and six months ended June 30, 2020, while revenues of $0.4 million and $1.1 million, respectively, were recognized for the comparable periods in 2019. License and contract revenues in 2019 resulted from royalty and other revenues recognized from Les Laboratoires Servier and Institut de Recherches Internationales Servier ("Servier") related to transition period activities pursuant to the terms of the Termination and Transfer Agreement with Servier.

Net loss for the three months ended June 30, 2020 was $14.0 million, or $(0.19) for basic and diluted loss per share, compared to net loss of $11.0 million, or $(0.19) for basic and diluted loss per share, for the same period in 2019. Net loss for the six months ended June 30, 2020 was $26.2 million, or $(0.38) for basic and diluted loss per share, compared to net loss of $21.8 million, or $(0.38) for basic and diluted loss per share, for the same period in 2019.

As of June 30, 2020, cash, cash equivalents and short-term investments totaled $70.1 million, compared to $33.7 million as of December 31, 2019. We expect current cash and cash equivalents will enable us to fund our operations into the fourth quarter of 2021.

On August 6, 2020Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported its financial results for the second quarter ended June 30, 2020 and provided a corporate update (Press release, Ziopharm, AUG 6, 2020, View Source [SID1234563088]).

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"During the second quarter, we were able to report positive clinical and organizational progress, positioning the Company for additional data readouts in the coming year," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer. "With the completion of enrollment in our phase 2 trial of Controlled IL-12 in combination with Libtayo, and launches of two new trials, we have begun to see encouraging signs of our partners re-emerging despite the COVID-19 outbreak."

Recent Corporate Highlights

Sleeping Beauty TCR-T Program

NCI Phase 2 Personalized TCR-T Trial. During the quarter and in recent weeks, the Ziopharm team collaborated with the National Cancer Institute (NCI) to advance manufacturing preparations for dosing the first patient in this first-in-human non-viral TCR-T study (NCT0402436). As NCI laboratory functions gradually re-open, the team there is once again proactively screening patients for neoantigens and T-cell receptors (TCRs) to render them eligible for the trial. Ziopharm has been able to use its newly expanded laboratories in Houston to complete required engineering runs and provided that information to the NCI to help expedite their enrollment to the trial.

Personalized and Library TCR-T Clinical Trials with MD Anderson. Based on interactions with the U.S. Food and Drug Administration (FDA), the Company continues to make progress toward finalizing the design of its TCR-T clinical trials at MD Anderson based on the Sleeping Beauty platform and clearance of an Investigational New Drug (IND) application to the FDA in Q1 2021. The Company plans to evaluate both its personalized TCR-T and its library TCR-T therapies and is focused on completing IND-enabling CMC (chemistry, manufacturing and controls) and nonclinical data package to support those opportunities. Given its progress assembling a TCR library, the Company expects the trial using these allogeneic receptors to begin enrollment first, with anticipated cancer indications of gynecologic, colorectal, pancreatic, non-small cell lung cancer and cholangiocarcinoma.

Controlled IL-12 Program

Phase 2 Combination Study. Ziopharm announced completion of enrollment in June of its phase 2 combination trial of Controlled IL-12 with Regeneron’s Libtayo to treat patients with recurrent glioblastoma (rGBM). Per the study protocol, 40 patients were enrolled at 7 sites, of whom 39 were dosed with the combination. Initial data is expected to be submitted for presentation later this year.

Pediatric Trial. The Company recently announced that the first patient with diffuse intrinsic pontine glioma (DIPG) had been dosed in its phase 1/2 study of Controlled IL-12 for the treatment of pediatric brain tumors. The trial (NCT03330197) is designed to evaluate the safety and tolerability of a single intratumoral injection of Ad-RTS-hIL-12 with up to 14 days of oral veledimex in children with gliomas. Up to 12 patients with DIPG may be enrolled in phase 1 of the study, which is being conducted at leading pediatric cancer centers across the United States, including Lurie Children’s Hospital in Chicago, the Dana-Farber Cancer Institute in Boston and the University of California in San Francisco.

ASCO 2020. The Company provided positive clinical updates at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) virtual annual meeting in late May. Continued follow-up from Controlled IL-12 monotherapy studies reinforced encouraging median overall survival, while Controlled IL-12 in combination with a PD-1 inhibitor demonstrated a favorable safety profile and encouraging initial survival data. Additional data are expected to be presented later this year.

Sleeping Beauty CAR-T Program

Eden BioCell CAR-T Study. The Company’s joint venture partner, Eden BioCell, continued to make progress toward filing an IND this year for a clinical trial in Taiwan to assess patient-derived (autologous) CD19-specific membrane bound IL-15 CAR-T cells, produced using Rapid Personalized Manufacturing (RPM) technology designed to reduce cost and simplify production for infusion the day after gene transfer.

Ziopharm CAR-T Study. The Company’s clinical collaborators at MD Anderson Cancer Center were able to complete necessary preparations and commence enrollment in the phase 1 clinical trial infusing donor-derived (allogeneic) CD19-specific CAR-T therapies produced using RPM. Up to 24 patients with advanced CD19+ leukemias and lymphomas who have relapsed after allogeneic bone marrow transplantation will be enrolled in this investigator-initiated trial (NCT03579888).

Operational

Expanded Board of Directors. Ziopharm recently announced the appointment of James Huang to the Company’s Board of Directors. Mr. Huang is a Managing Partner at Kleiner Perkins Caufield & Byers China and has founded and financed several innovative life sciences companies, including GenScript, Legend Biotech and Zai Lab. He is also Founding Partner of Panacea Venture, which formed TriArm Therapeutics, the funding partner for Ziopharm’s joint venture, Eden BioCell.

Scientific Advisory Board. In June, the Company announced the appointment of renowned oncology and immunotherapy pioneer, Carl June, M.D., as Chairman of its newly formed Scientific Advisory Board (SAB). Dr. June is recognized in the oncology field for his groundbreaking work in the development and commercialization of gene therapy and T-cell therapies. The SAB will provide strategic counsel to guide the efficient development of Ziopharm’s innovative technologies and pipeline of immunotherapies.

Second Quarter 2020 Financial Results

Research and development expenses were $12.1 million for the second quarter of 2020, compared to $10.0 million for the second quarter of 2019, primarily reflecting increased clinical trial activity.

General and administrative expenses were $6.6 million for the second quarter of 2020, compared to $4.8 million for the second quarter of 2019. The increase in general and administrative expenses for the second quarter of 2020 is primarily due to increased headcount, legal costs associated with its expanded patent portfolio and facility costs.

Net loss for the second quarter of 2020, was $18.6 million, or $(0.09) per share, compared to a net loss of $14.6 million, or $(0.09) per share, for the second quarter of 2019.

Cash and cash equivalents, as of June 30, 2020 were $153.5 million.

A prepayment of approximately $14.0 million remains for work to be conducted by the Company at MD Anderson under the Company’s research and development agreements.

Conference Call and Webcast

Ziopharm will host a conference call and webcast for the investment community today, August 6, 2020, at 4:30 p.m. EDT. The conference call can be accessed by dialing 1-877-451-6152 (U.S. and Canada) or 1-201-389-0879 (international). The passcode for the conference call is 13706729. To access the live webcast or the subsequent archived recording, click here or visit the "Investors" section of the Ziopharm website at www.ziopharm.com. The webcast will be recorded and available for replay on the company’s website for two weeks.

On August 6, 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 second quarter ended June 30, 2020 (Press release, Lineage Cell Therapeutics, AUG 6, 2020, View Source [SID1234563087]). Lineage management will host a conference call today at 5:30 p.m. Eastern Time/2:30 p.m. Pacific Time to discuss its second quarter 2020 financial and operating results and to provide a business update.

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"Lineage has continued to make significant progress across our entire cell therapy pipeline, with positive milestones on each of our three programs announced this quarter," stated Brian M. Culley, Lineage CEO. "Most importantly, after moving into a cohort of patients with less advanced disease, we reported the first known finding of retinal tissue restoration, which we believe could become a landmark discovery in the treatment of dry AMD. Specifically, Lineage reported evidence of substantial anatomical restoration of retinal tissue within an area of geographic atrophy, or GA. The area of GA was approximately 25% smaller when assessed at 9 months, compared to the patient’s pre-treatment baseline. These findings were initially discovered by an independent external advisor using multiple imaging technologies and were subsequently confirmed by the reading center and additional experts in the field of retinal imaging. In light of the significance of this finding, our objective is to treat and monitor the final three patients in Cohort 4 of our dry AMD study and seek to demonstrate additional evidence of this exciting finding. These data also will direct our clinical, regulatory, and ongoing partnership discussions.

We also recently exercised an option with Cancer Research UK, which allowed us to assume responsibility for advancing our dendritic cell vaccine program, strengthening our pipeline and moving Lineage more prominently into the field of cancer immunotherapy. The VAC platform will permit us to expand not only our oncology pipeline, but also enter infectious diseases, with steps being taken to identify external funding to develop a SARS-CoV-2 vaccine, based on VAC platform data showing strong induction of cellular immunity via T cells.

Lastly, we have made significant progress developing and implementing manufacturing improvements for our OPC1 program to treat spinal cord injuries. We now are working to develop a "thaw-and-inject" formulation and are evaluating a superior delivery system to enable an easier and faster surgical procedure and facilitate enrollment in a late-stage clinical study.

Our aim is to provide safe and sustained benefits of targeted cell-based therapies, alongside commercially relevant solutions with competitive advantages in areas of scale-up, production costs, and delivery techniques. We believe our holistic approach to designing new therapeutic solutions will allow us to position ourselves as the clear leader in the emerging field of cell therapy transplant medicine."

Lineage has the following plans and objectives for the second half of 2020:

-Meet with Biomedical Advanced Research and Development Authority (BARDA) in August to discuss our proposal for non-dilutive support for a coronavirus vaccine candidate.

-Report initial VAC2 clinical data from patients treated in the ongoing Phase 1 trial in NSCLC (non-small cell lung cancer) run by Cancer Research UK.

-Complete patient enrollment in the U.S. with the Gyroscope Orbit SDS and new thaw-and-inject formulation in the ongoing Phase 1/2a clinical trial of OpRegen for the treatment of dry AMD.

-Present new and accumulated OpRegen data from the ongoing Phase 1/2a clinical trial at the American Academy of Ophthalmology (AAO) Annual 2020 Meeting the second week of November.

-Meet with the U.S. Food and Drug Administration (FDA) to discuss further development of the OPC1 program.

Balance Sheet and Cash Flow Highlights

Cash, cash equivalents, and marketable securities totaled $20.3 million as of June 30, 2020. Marketable securities include our remaining ownership of unrestricted securities in OncoCyte Corporation (OncoCyte), AgeX Therapeutics, Inc. (AgeX) and Hadasit Bio-Holdings Ltd (Hadasit).

We have continued to fund our operations primarily by selling a portion of our marketable securities. In the six months ended June 30, 2020, we sold approximately 4.8 million shares of OncoCyte common stock for net proceeds of $10.9 million. We continue to hold approximately 3.6 million shares of OncoCyte stock that are valued at $6.8 million as of August 4, 2020, based on the closing price of its common stock on that date. 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 later this month, on August 30, 2020. As of June 30, 2020, the outstanding principal and accrued interest on the note was $24.4 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.

Net cash used in operating activities for the six months ended June 30, 2020 was approximately $9.3 million, a decrease of $9.7 million as compared to $19.0 million in the same period of 2019. Additionally, net cash used in the three months ended June 30, 2020 of $4.3 million is $0.7 million lower than net cash used in the three months ended March 31, 2020 of $5.0 million.

Second Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from research grants, royalties and licensing fees. Total revenues for the three months ended June 30, 2020 were $0.4 million, a decrease of $0.4 million as compared to $0.8 million for the same period in 2019. The decrease was primarily related to a $0.2 million decrease in grant revenue due to the timing of grant related activities for OpRegen and other ophthalmic applications and a $0.1 million decrease in the sale of research products and services due to the cessation of such sales.

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 June 30, 2020 were approximately $6.7 million, a decrease of $4.8 million as compared to $11.5 million for the same period in 2019.

R&D Expenses: R&D expenses for the three months ended June 30, 2020 were $2.8 million, an approximate decrease of $2.4 million as compared to $5.2 million for the same period in 2019. The overall decrease was primarily related to decreases of $1.7 million in OpRegen and other ophthalmic application expenses, attributable primarily to a decrease in manufacturing activities in 2020 as compared to 2019, and $0.7 million in OPC1 expenses, primarily related to a decrease in development activities in 2020 as compared to 2019 when technology transfer was a focus to support OPC1 coming in-house with the acquisition of Asterias.

G&A Expenses: G&A expenses for the three months ended June 30, 2020 were $3.9 million, a decrease of $2.4 million as compared to approximately $6.3 million for the same period in 2019. The decrease was primarily attributable to a $1.6 million reduction in expenses related to our merger with Asterias Biotherapeutics, Inc. (Asterias), a $0.2 million reduction in compensation expenses, a $0.2 million reduction in investor and public relations expenses, a $0.2 million reduction in accounting expenses, a $0.1 million reduction in travel expenses, a $0.1 million reduction in rent expenses and a $0.1 million reduction in consulting expenses, offset by a $0.2 million increase related to the cessation of shared services reimbursements.

Loss from Operations: Loss from operations for the three months ended June 30, 2020 was $6.4 million, a decrease of $4.4 million as compared to $10.8 million for the same period in 2019.

Other (Expense) Income, Net: Other income/(expenses), net for the three months ended June 30, 2020 reflected other expense, net of ($0.1) million, compared to other expense, net of ($20.5) million for the same period in 2019. The variance was primarily related to changes in the value of equity method investments and marketable equity securities for the applicable periods, as well as foreign currency translation adjustments related to Lineage’s international subsidiaries. The value of Lineage’s OncoCyte shares decreased by $21.4 million in the three months ended June 30, 2019, which contributed greatly to the overall balance in other expense, net for that period.

Net loss attributable to Lineage: The net loss attributable to Lineage for the three months ended June 30, 2020 was $6.5 million, or $0.04 per share (basic and diluted), compared to a net loss attributable to Lineage of $30.0 million, or $0.20 per share (basic and diluted), for the same period in 2019.

Conference Call and Webcast

Lineage will host a conference call and webcast today, at 2:30 pm PT/5:30 pm ET to discuss its second quarter 2020 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 August 14, 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 6649516.