On August 6, 2020 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported financial results for the second quarter ended June 30, 2020 (Press release, Geron, AUG 6, 2020, View Source [SID1234563094]). The Company will host a conference call today at 4:30 p.m. ET to discuss second quarter financial results and current events.

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"In the second quarter, we achieved a number of key milestones that have changed the trajectory of the Company," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "After a successful meeting with the FDA, we announced plans to move forward with a Phase 3 clinical trial in refractory MF with overall survival as the primary endpoint. This decision was based on overall survival data from the IMbark Phase 2 trial that suggest imetelstat treatment could potentially double the remaining life expectancy for patients who have become refractory to JAK inhibitors."

Dr. Scarlett added, "We reported clinically meaningful transfusion independence with longer durability from more mature data in the IMerge Phase 2 trial in lower risk MDS, which included 29% of patients being transfusion-free for more than one year. We also raised additional capital in the second quarter that we expect will sufficiently fund our development plans for the ongoing Phase 3 IMerge trial in lower risk MDS and the upcoming Phase 3 trial in refractory MF into the second half of 2022. These achievements, along with our experienced development team, position Geron to become a leader in the treatment of hematologic myeloid malignancies over the next several years."

Current Events – Clinical Development and Regulatory

Ongoing IMerge Phase 3 Clinical Trial in Myelodysplastic Syndromes (MDS)

As of the end of July 2020, approximately 93% of the 90 clinical sites originally planned for the trial were open for screening and enrollment, compared to 68% in May.

The momentum of patient enrollment has begun to improve as the effects of the COVID-19 pandemic begin to wane in a majority of the countries where IMerge clinical sites are located. The Company continues to expect patient enrollment to be completed by the end of the first quarter of 2021, subject to potential future delays or interruptions associated with COVID-19. To help ensure achievement of this goal, the trial is being expanded from 90 to approximately 130 clinical sites. Geron expects a majority of these 40 new sites to be open for enrollment by the end of the year. Under current enrollment assumptions, the Company continues to expect top-line results in the second half of 2022.

Start-Up Activities for Planned Phase 3 Clinical Trial in Myelofibrosis (MF)

Geron expects to engage over 150 sites across North America, South America, Europe and Asia to participate in the planned global Phase 3 clinical trial in refractory MF. Trial start-up activities underway include identifying potential clinical sites for participation, as well as finalizing the clinical trial protocol. Geron plans to open the trial for screening and enrollment in the first quarter of 2021. Under current planning assumptions, Geron expects to complete patient enrollment in the second half of 2022, to conduct an interim analysis in the first half of 2023 and to conduct a final analysis in the first half of 2024.

European Commission Grants Orphan Drug Designation to Imetelstat for MDS

At the end of July, the European Commission granted orphan drug designation to imetelstat as a potential treatment for MDS based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. Imetelstat has already been granted orphan drug designation by the United States Food and Drug Administration as a potential treatment for MDS.

Geron Corporation | Confidential and Proprietary

Recent Data Presentations in June

KOL Event Highlighted Compelling Phase 2 Data from the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress

Key opinion leaders within hematologic myeloid malignancies reprised four presentations containing new clinical data and analyses related to imetelstat from the Virtual Edition of the EHA (Free EHA Whitepaper) Annual Congress.

The recent EHA (Free EHA Whitepaper) presentation of more mature data from 38 patients in the IMerge Phase 2 clinical trial in lower risk MDS reported notable continued durability, including a median duration of 8-week transfusion independence of 20 months, which is the longest duration reported to date in this trial. Also reported for the first time, 29% of the 38 patients were transfusion free for more than one year.

In addition, new analyses from the IMbark Phase 2 clinical trial in relapsed/refractory MF correlated the median overall survival observed in the trial with other clinical endpoints, such as improvement in fibrosis. These analyses provide further support for the planned Phase 3 clinical trial in refractory MF.

Overall, the EHA (Free EHA Whitepaper) data and analyses continue to support the potential disease-modifying activity with imetelstat treatment as evidenced by the clinically meaningful durable transfusion independence in the Phase 2 IMerge trial and the improvement in overall survival in the Phase 2 IMbark trial.

Strengthened Balance Sheet to Achieve Development Plans

As of June 30, 2020, Geron had approximately $265 million in cash, cash equivalents, and current and non-current marketable securities. The Company’s cash position reflects net proceeds of approximately $140 million from a public offering of securities in the second quarter. Based on current planning assumptions, the Company expects such funds to be sufficient for its operations into the second half of 2022 when top-line results for the IMerge Phase 3 clinical trial in lower risk MDS and completion of patient enrollment for the planned Phase 3 clinical trial in refractory MF are expected.

Second Quarter and Year-to-Date 2020 Results

For the second quarter of 2020, the Company reported a net loss of $15.8 million, or $0.06 per share, compared to $14.2 million, or $0.08 per share, for the comparable 2019 period. Net loss for the first six months of 2020 was $32.2 million, or $0.14 per share, compared to $24.3 million, or $0.13 per share, for the comparable 2019 period.

Revenues for the three and six months ended June 30, 2020 were $43,000 and $95,000, respectively, compared to $101,000 and $158,000 for the comparable 2019 periods. Revenues in 2020 and 2019 primarily reflect estimated royalties from sales of cell-based research products from the Company’s divested stem cell assets. In connection with the divestiture of Geron’s human embryonic stem cell assets, including intellectual property and proprietary technology, to Lineage Cell Therapeutics, Inc. (formerly BioTime, Inc., which acquired Asterias Biotherapeutics, Inc.) in 2013, Geron is entitled to receive royalties on future product sales.

Total operating expenses for the three and six months ended June 30, 2020 were $16.8 million and $33.7 million, respectively, compared to $15.3 million and $26.7 million for the comparable 2019 periods.

Research and development expenses for the three and six months ended June 30, 2020 were $10.8 million and $21.6 million, respectively, compared to $10.1 million and $16.0 million for the comparable 2019 periods. The increase in research and development expenses for the three and six months ended June 30, 2020, compared to the same periods in 2019, primarily reflects higher clinical development costs associated with the IMerge Phase 3 clinical trial, increased costs in connection with validating the imetelstat manufacturing process at contract manufacturers and higher personnel-related costs for additions to the development team in 2019.

Geron Corporation | Confidential and Proprietary

General and administrative expenses for the three and six months ended June 30, 2020 were $6.0 million and $12.1 million, respectively, compared to $5.2 million and $10.6 million for the comparable 2019 periods. The increase in general and administrative expenses for the three and six months ended June 30, 2020, compared to same periods in 2019, primarily reflects increased personnel-related expenses for additional general and administrative headcount to support growing operational activities.

Interest and other income for the three and six months ended June 30, 2020 was $475,000 and $1.2 million, respectively, compared to $1.1 million and $2.3 million for the comparable 2019 periods. The decrease in interest and other income for the three and six months ended June 30, 2020, compared to same periods in 2019, primarily reflects lower yields on the Company’s reduced marketable securities portfolio.

2020 Financial Guidance Reaffirmed

The Company expects its 2020 operating expense burn to range from $70 to $75 million. This guidance reflects cash conservation measures implemented in April due to the COVID-19 pandemic, such as suspending travel and postponing a planned imetelstat proof of concept study. It also reflects new costs for start-up activities associated with the planned Phase 3 clinical trial in refractory MF and additional costs for the expansion of clinical sites for the IMerge Phase 3 clinical trial.

Conference Call

Geron will host a conference call at 4:30 p.m. ET on Thursday, August 6, 2020 to discuss second quarter financial results and recent events.

A live, listen-only webcast will be available on the Company’s website at www.geron.com/investors/events. If you are unable to listen to the live call, an archived webcast will be available on the Company’s website for 30 days.

Participants may access the conference call live via telephone by dialing domestically +1 (888) 869-1189 or internationally +1 (706) 643-5902. The conference ID is 8496138. To minimize potential registration and access delays, Geron has implemented Direct Event, which allows participants to pre-register online using the following link, View Source Upon registration, a Direct Event Passcode and unique Registrant ID will be sent via email and will be needed in order to enter the conference call. Participants are advised to pre-register at least 10 minutes prior to joining the call.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the apoptosis of malignant stem and progenitor cells, which allows potential recovery of normal hematopoiesis. Geron’s imetelstat development program includes two registration-enabling studies, IMerge, an ongoing Phase 2/3 clinical trial in lower risk myelodysplastic syndromes (MDS), and a planned Phase 3 clinical trial in refractory myelofibrosis (MF) expected to be open for patient screening and enrollment in the first quarter of 2021. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

On August 6, 2020 Navidea Biopharmaceuticals, Inc. (NYSE American:NAVB) ("Navidea" or the "Company"), a company focused on the development of precision immunodiagnostic agents and immunotherapeutics, reported it will host a conference call and webcast on Thursday, August 13, 2020 at 5:00 p.m. (EDT) to discuss financial results and corporate developments for the second quarter ended June 30, 2020 (Press release, Navidea Biopharmaceuticals, AUG 6, 2020, View Source [SID1234563093]).

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Jed Latkin, Chief Executive Officer, Dr. Michael Rosol, Chief Medical Officer, and Erika Eves, Director of Finance and Administration, will host the call and webcast to discuss the financial results and provide an update on recent developments and clinical progress. Management will be available to answer questions live immediately following the earnings announcement and prepared remarks portion of the call.

To participate in the call and webcast, please refer to the information below:

Event: Q2 2020 Earnings and Business Update Conference Call

Date: Thursday, August 13, 2020

Time: 5:00 p.m. (EDT)

U.S. & Canada Dial-in: 877-407-0312

International Dial-in: +1 201-389-0899

Conference ID: 13708190

Webcast Link: View Source

A live audio webcast of the conference call will also be available on the investor relations page of Navidea’s corporate website at www.navidea.com. In addition, the recorded conference call can be replayed and will be available for 90 days following the call on Navidea’s website.

On August 6, 2020 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody technology platform, reported second quarter 2020 financial results and provided a business update (Press release, CytomX Therapeutics, AUG 6, 2020, View Source/news-releases/news-release-details/cytomx-therapeutics-announces-second-quarter-2020-financial" target="_blank" title="View Source/news-releases/news-release-details/cytomx-therapeutics-announces-second-quarter-2020-financial" rel="nofollow">View Source [SID1234563092]).

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"CytomX made broad progress across our clinical and preclinical programs during the second quarter as we further advanced our technology platform, partnerships, and lead drug candidates to several important inflection points," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "We continue to show leadership in defining new therapeutic antibody modalities with demonstrated potential to address significant unmet medical needs in the treatment of cancer, a mission that remains as important as ever despite the unprecedented challenges being posed by the COVID-19 pandemic."

SECOND QUARTER BUSINESS HIGHLIGHTS AND RECENT DEVELOPMENTS

Clinical Pipeline Progress: ASCO (Free ASCO Whitepaper)20 Data Presentations and Program Next Steps

In May, CytomX presented broad clinical pipeline progress at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program from four Probody programs advancing in, or towards, Phase 2 studies:

CX-2029 and CX-2009, Probody Drug Conjugates designed to target the previously undruggable targets, CD71 and CD166, respectively
BMS-986249, a Probody version of the anti-CTLA-4 immunotherapeutic antibody, ipilimumab (Yervoy) and
CX-072, a Probody immunotherapeutic targeting PD-L1.
CX-2029 Phase 1 Data: Preliminary Validation of CD71 as a Novel Oncology Target and Advancement to Phase 2 Expansion Cohorts

CytomX and its partner AbbVie presented preliminary clinical data from the first-in-human, Phase 1 dose-escalation study of CX-2029, a Probody drug conjugate targeting the previously undruggable target, CD71 (transferrin receptor), in patients with solid tumors.
Evidence of target lesion reduction was seen at doses of ≥2 mg/kg including confirmed partial responses in squamous non-small cell lung cancer (sqNSCLC) and squamous head and neck cancer (HNSCC), which were observed at 3 mg/kg.
CX-2029 was generally well tolerated with anemia and infusion-related reactions being the most common adverse events, supporting 3 mg/kg as the Phase 2 expansion dose.
Phase 2 expansion cohorts are expected to be initiated in the second half of 2020 in patients with sqNSCLC, HNSCC, esophageal cancer, and diffuse large B-cell lymphoma.
CX-2009: Phase 2 Strategies Focused in HER2 Negative Breast Cancer Subtypes

CytomX presented updated clinical data from the first-in-human, dose-escalation, monotherapy Phase 1 study of CX-2009, a Probody drug conjugate targeting the previously undruggable target, CD166, in seven selected tumor types.
Evidence of target lesion reduction was observed at doses of ≥4 mg/kg including confirmed and unconfirmed partial responses in patients with HER2- breast cancer.
CX-2009 was generally well tolerated at doses up to 7 mg/kg, the dose selected for Phase 2 expansion studies.
The previously initiated Phase 2 expansion study of CX-2009 study in HER2 negative/hormone receptor positive (HER2-/HR+) breast cancer was paused in March 2020 due to the impact of the COVID-19 pandemic. In the intervening period, this study strategy has been revised to further focus patient enrollment criteria and is expected to be re-initiated during the second half of 2020. The revised Phase 2 study will continue to evaluate CX-2009 as monotherapy at 7 mg/kg administered every three weeks and enroll at least 40 patients.
CytomX also expects to initiate a Phase 2 expansion study during the second half of 2020 evaluating CX-2009 as monotherapy and in combination with CX-072, the Company’s anti-PD-L1 Probody therapeutic candidate, in patients with triple negative breast cancer (TNBC).
BMS-986249: Anti-CTLA-4 Probody Immunotherapeutic

Bristol Myers Squibb presented safety data from the dose escalation stage of a Phase 1/2a trial of BMS-986249, a Probody version of the anti-CTLA-4 antibody ipilimumab.
This trial assessed the safety, pharmacokinetics, and pharmacodynamics of escalating doses of BMS-986249 as monotherapy or in combination with the anti PD-1 antibody nivolumab (Opdivo) in patients with advanced cancers. The doses of BMS-986249 ranged from 240 mg to 2400 mg (approximately 3 – 30 mg/kg).
BMS-986249 was generally well tolerated as monotherapy and in combination with nivolumab. The types of treatment-related adverse events were consistent with CTLA-4 blockade, and the overall data align with the proposed Probody mechanism of action. No new safety signals were observed.
Bristol Myers Squibb has initiated the Part 2a randomized cohort expansion of the ongoing Phase 1/2a trial of BMS-986249 in combination with nivolumab in patients with metastatic melanoma.
During the second quarter, Bristol Myers Squibb also presented a comprehensive preclinical dataset from the anti-CTLA-4 Probody immunotherapeutics, BMS-986249 and BMS-986288, a non-fucosylated Probody of ipilimumab, at the American Association of Cancer Research’s (AACR) (Free AACR Whitepaper) 2020 Virtual Annual Meeting II. These data support the strategy of expanding the therapeutic index for CTLA-4 therapy using CytomX’s Probody technology and provides rationale for the ongoing clinical studies of these agents.

CX-072: Anti-PD-L1 Probody Immunotherapeutic

CytomX presented data from seven expansion cohorts evaluating CX-072, an anti-PD-L1 Probody therapeutic administered at 10 mg/kg. CX-072 demonstrated durable anti-tumor activity in patients with IO-sensitive tumors such as TNBC, anal squamous cell carcinoma, cutaneous squamous cell carcinoma, and tumors with high mutational burden.
Grade 3/4 TRAEs were 10% and 5.9% for those patients who received monotherapy < 6 months and ≥ 6 months, respectively. Long term patients experienced fewer immune-related adverse events (irAEs) and had no grade 3+ irAEs.
The clinical profile of CX-072 continues to be consistent with this agent being a differentiated combination therapy partner. CX-072 will next be evaluated in combination with CX-2009 in TNBC.
Preclinical Pipeline Progress

CX-904 EGFR-CD3 Probody Bispecific

CytomX continued to advance CX-904, the lead candidate from the Epidermal Growth Factor Receptor-CD3 T-Cell Bispecific program, towards IND-enabling studies. CX-904 is partnered with Amgen as part of a global co-development agreement.
CX-2043 EpCAM Probody Drug Conjugate

CytomX continued to advance CX-2043, the lead candidate from the epithelial cell adhesion molecule (EpCAM)-targeting Probody Drug Conjugate program, towards IND-enabling studies.
Astellas Collaboration

CytomX launched discovery activities within the Astellas strategic collaboration that was announced in the first quarter and focused on the discovery, research, development, and commercialization of novel T-cell engaging bispecific antibodies.
COVID-19 Pandemic and Business Continuity

CytomX is committed to ensuring the health, safety and well-being of its clinical study participants, study site staff, and our employees. CytomX continues to closely monitor the COVID-19 pandemic situation and is following local, state, and federal guidelines, including emerging Health Authority guidance and IRB/Ethics Committee recommendations with respect to the conduct of our worldwide clinical trials. In accordance with state and local guidelines, CytomX is following a work-from-home protocol for many of its employees with only select staff, including those in research functions that require laboratory access, having access to CytomX’s corporate offices where multiple layers of safety measures have been put in place.

Second Quarter 2020 Financial Results

Cash, cash equivalents and short-term investments totaled $346.4 million as of June 30, 2020, compared to $296.1 million as of December 31, 2019.

Revenue was $16.6 million for the three months ended June 30, 2020, compared to $9.0 million for the three months ended June 30, 2019. The net increase in revenue of $7.6 million was primarily due to an increase of $3.4 million from the Amgen EGFR project as a result of a higher percentage of completion progress in the second quarter of 2020, and an increase of $4.2 million related to the recognition of revenue from the $80 million upfront payment under the Collaboration and License Agreement with Astellas entered into in March 2020.

Research and development expenses decreased by $6.8 million during the three months ended June 30, 2020 compared to that in the corresponding period in 2019. The decrease was largely attributed to $3.4 million for the University of California, Santa Barbara (UCSB) fees paid in 2019 relating to the amendment to the license agreement, and a $0.8 sublicense fee also paid to UCSB in 2019 relating to a $10.0 million milestone earned by the Company for AbbVie’s selection of a second target under the Amended and Restated Discovery Collaboration and License Agreement with AbbVie in 2019 and a decrease of $2.3 million in clinical related expenses due to the decrease in clinical trial activities.

General and administrative expenses decreased by $0.7 million during the three months ended June 30, 2020 compared to that in the corresponding period in 2019 primarily due to a decrease in stock base compensation expense.

Teleconference Scheduled Today at 5:30 p.m. ET
Conference Call/Webcast Information

CytomX management will host a conference call today at 5:30 p.m. ET. Interested parties may access the live audio webcast of the teleconference through the "Investor & News" section of CytomX’s website at View Source or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) and using the passcode 5399347. An archive of the webcast will be available on the CytomX website from August 6, 2020, until August 20, 2020.

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.