On November 12, 2019 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported third quarter 2019 financial results and business updates, which will be discussed on a conference call scheduled for Wednesday, November 13 at 10:00 a.m. ET (Press release, Abeona Therapeutics, NOV 12, 2019, View Source [SID1234550984]). Interested parties are invited to participate in the call by dialing 844-602-0380 (toll-free domestic) or 862-298-0970 (international) or via webcast at View Source

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"Abeona has made progress on our lead clinical program, EB-101, for the treatment of recessive dystrophic epidermolysis bullosa," said João Siffert, M.D., Chief Executive Officer of Abeona. "We continue to anticipate receiving CMC clearance for this pivotal trial by year-end following the recent submission of additional transport stability data for EB-101, in response to the Clinical Hold Letter received in the quarter, as well as the submission of updated clinical and comparability protocols for the VIITALTM Phase 3 study."

Dr. Siffert continued, "We were also very pleased with the publication of positive long-term data from our Phase 1/2a clinical trial, reinforcing the potential of EB-101 to safely provide durable healing for the most challenging to treat, large and painful RDEB wounds. Three years post treatment, the majority of EB-101-treated wounds remained healed and without pain. We believe that EB-101 is uniquely positioned to address the needs of the majority of RDEB patients who suffer from these types of chronic wounds and we remain dedicated to delivering this therapy to the community."

Third Quarter Financial Results:

Cash, cash equivalents and marketable securities as of September 30, 2019, were $47.9 million compared to $62.5 million as of June 30, 2019. The decrease in cash was driven primarily by the net cash used in operating activities of $18.3 million.

Research and development expenses for the third quarter ended September 30, 2019 were $10.9 million compared to $13.2 million in the same period of 2018. The decrease in R&D expense was primarily attributable to decreased clinical and development work, partially offset by increased salary and related costs from the hiring of additional clinical, regulatory, manufacturing and quality staff.

General and administrative expenses for the third quarter ended September 30, 2019 were $4.7 million compared to $5.0 million in the same period of 2018. The decrease in G&A expenses was primarily due to decreased rental, recruiting, professional fee and salary related costs.

Net loss was $0.35 per share for the third quarter of 2019 compared to $0.34 per share in the same period of 2018.

Third Quarter and Recent Highlights:

●Submitted additional transport stability data for EB-101 in response to September 17 FDA Clinical Hold Letter regarding the planned Phase 3 VIITAL study
Submitted Phase 3 VIITAL clinical trial protocol with updated PRO assessments, and submitted the retrovirus comparability protocol to FDA.
●Presentation of data from the Transpher A Study, the Company’s ongoing Phase 1/2 clinical trial evaluating ABO-102 for the treatment of MPS IIIA, and research updates from its library of novel AIMTM adeno-associated virus capsids at the 27th European Society of Gene and Cell Therapy Congress.
●Publication of positive long-term safety and efficacy data from the Phase 1/2a clinical trial of EB-101 in JCI Insight with collaborators from Stanford University School of Medicine.
●Three years after treatment with EB-101, a majority of RDEB patients had sustained wound healing, with 80% (16/20) of wounds achieving ≥50% healing, and 70% (14/20) achieving ≥75%
●Two years after treatment, only 1 of 6 untreated (17%), prospectively selected control wounds, had ≥50% healing
●50% or greater wound healing was associated with no pain (0/16) and no itch (0/16) at treated sites three years post-treatment, compared with presence of pain in 53% (20/38) and itch in 61% (23/38) of wound sites at baseline
●EB-101 was associated with long-term molecular expression of type VII collagen protein, which plays an important role in anchoring the dermal and epidermal layers of the skin
●No serious treatment-related adverse events were observed during the three-year observation period
●Retained Jefferies LLC as its financial advisor to assist with the review of strategic intiatives focused on advancing the Company’s mission and maximizing stakeholder value.

On November 12, 2019 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on developing curative cell therapies for cancer, reported financial results and corporate updates for the third quarter ended September 30, 2019, and provided recent activities (Press release, Unum Therapeutics, NOV 12, 2019, View Source [SID1234550983]).

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"Our recently announced strategic focus towards addressing the challenge of treating solid tumor cancers is well underway with ACTR707, BOXR1030, and our BOXR platform that is designed to discover new product candidates aimed at improving the function of T cell therapies in the solid tumor microenvironment," said Chuck Wilson Ph.D., President and Chief Executive Officer of Unum. "BOXR1030, which co-expresses the GOT2 transgene and is designed to improve T cell metabolism and reduce T cell exhaustion, generated complete tumor regressions under metabolically challenging conditions in preclinical studies as presented at the SITC (Free SITC Whitepaper) meeting. For ACTR707, our Phase 1 trial is progressing nicely and we remain on track to report enrollment and early safety updates from patients treated in the first dose cohort by the end of this year."

Recent Program and Corporate Highlights

Announced strategic focus on developing best-in-class cellular therapies for solid tumor cancers: Unum is uniquely positioned to address the challenge of treating solid tumor cancers with its two platforms—ACTR and BOXR—having recently validated ACTR in the hematologic setting and with preclinical data recently emerging from BOXR1030, the first product candidate from BOXR. Unum’s ACTR platform enables selective T cell targeting for on-tumor attack while its BOXR platform is designed to improve T cell functionality in the solid tumor microenvironment (TME) through the co-expression of novel transgenes. Unum’s priorities in solid tumors include: 1) completing the ongoing ATTCK-34-01 Phase 1 trial of ACTR707 in HER2+ cancers, 2) advancing BOXR1030 towards the clinic with an anticipated IND filing in late 2020; and 3) expanding its BOXR platform to accelerate discovery of new product candidates across a broad range of immune cell therapies, including both autologous and allogeneic approaches. Five clinical trial sites are activated and Unum expects to report preliminary safety data from patients treated in the first dose cohort of the Phase 1 ATTCK-34-01 trial of ACTR707 combined with trastuzumab to treat advanced HER2+ solid tumor cancers by the end of this year, and to report safety and clinical response data from multiple dose cohorts in 2020.

Presented preclinical data for BOXR1030 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting (November 6-10): BOXR1030 expresses a glypican-3 (GPC3) targeted chimeric antigen receptor (CAR) with the addition of the "bolt-on" transgene glutamic-oxaloacetic transaminase 2 (GOT2) to improve T cell function in the TME by enhancing T cell metabolism. As presented at the SITC (Free SITC Whitepaper) conference, expression of the GOT2 mitochondrial enzyme in BOXR1030 increased the production of key amino acids and metabolites, improved the anti-oxidant balance of T cells, and prevented their dysfunction and exhaustion in preclinical studies using stringent animal xenograft models that simulate the TME. In vitro, BOXR1030 T cells were resistant to suppressive TME-like conditions, showing improved T cell proliferation under both hypoxic and low glucose conditions compared with control GPC3+ CAR-T cells. In vivo, BOXR1030 demonstrated superior activity compared to the control CAR-T with treated animals achieving complete tumor regressions under metabolically challenging conditions. Tumor infiltrating lymphocytes isolated from the tumors of treated animals revealed that BOXR1030 cells were more resistant to dysfunction, had fewer markers of exhaustion, and remained functional as compared to the control CAR-T cells.

Announced de-prioritized investment in hematologic programs. The clinical response and tolerability data recently generated from the ATTCK-20-03 Phase 1 trial in non-Hodgkin lymphoma established proof-of-concept for ACTR707, allowing the company to focus its efforts towards the ATTCK-34-01 Phase 1 trial in solid tumors, a clinical setting for which ACTR707 was originally developed. With favorable tolerability at relatively low doses explored to date, Unum announced plans to continue limited dose escalation to inform potential future development of the program in 2020.
Separately, Unum and its partner, Seattle Genetics, Inc., have suspended further dose-escalation of the ATTCK-17-01 Phase 1 trial of ACTR087 with SEA-BCMA in multiple myeloma pending a further review of this program. Two additional cohorts of patients have been treated in the Phase 1 trial in 2019, escalating doses of the SEA-BCMA antibody to 2.0 mg/kg and of the ACTR087+ T cells to 50M. No dose-limiting toxicities (DLTs) following ACTR087 administration were reported and no severe adverse events of cytokine release syndrome (CRS) or neurologic events have been observed to date.
Announced accepted oral and poster presentations at upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, December 7-10, Orlando, FL.

Title: "A Phase 1 Study of ACTR087 in Combination with Rituximab, in Subjects with Relapsed or Refractory CD20-Positive B-Cell Lymphoma"
Presenting Author: Javier Munoz, M.D., M.S., Banner MD Anderson Cancer Center, Gilbert AZ
Date & Time: Oral #244, Saturday, December 7, 2019, 2:45 p.m. ET

Title: "Preliminary Clinical Results from a Phase 1 Study of ACTR707 in Combination With Rituximab in Subjects with Relapsed or Refractory CD20+ Non-Hodgkin Lymphoma"
Presenting Author: Ian Flinn, M.D., Ph.D., Sarah Cannon Research Institute, Nashville, TN
Date & Time: Poster #1587, Saturday, December 7, 2019, 5:30 p.m. ET
Third Quarter 2019 Financial Results

Collaboration Revenue: Collaboration revenue recognized during the third quarter ended September 30, 2019 was $1.0 million, compared to $2.0 million in the same period of 2018. Collaboration revenue, which includes the recognition of a portion of the upfront payment received from Seattle Genetics, Inc. as well as reimbursements of research and development costs attributed to the Seattle Genetics, Inc. collaboration agreement, decreased as a result of fewer activities related to the programs under the collaboration agreement.

R&D Expenses: Research and development expenses were $10.3 million for the third quarter ended September 30, 2019 compared to $10.3 million for the same period of 2018. Research and development expenses relate to costs for the ongoing Phase 1 trials and preclinical programs, as well as personnel-related costs to support these programs.

G&A Expenses: General and administrative expenses for the third quarter ended September 30, 2019 were $2.7 million, compared to $2.4 million for the same period of 2018. The increase is primarily related to increased headcount and personnel-related costs as well as expenses required to operate as a public company.

Net Loss: Net loss attributable to common stockholders was $12.0 million, or $0.39 per share, for the third quarter ended September 30, 2019 compared with a net loss attributable to common stockholders of $10.2 million, or $0.34 per share, for the same period of 2018.

Cash and Cash Equivalents: As of September 30, 2019, Unum had cash and cash equivalents of $45.9 million. Unum believes that its existing cash and cash equivalents will fund operating expenses and capital expenditure requirements into early 2021.

On November 12, 2019 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported recent business highlights, anticipated upcoming milestones, and financial results for the third quarter and first nine months of 2019 (Press release, BeiGene, NOV 12, 2019, View Source [SID1234550982]).

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"We recently announced a transformative collaboration with Amgen that we expect to close in early 2020, at which point we’ll begin executing on the commercialization and development plans for Amgen’s three commercial-stage drugs and 20 drug candidates in China. We believe that this collaboration fortifies our position as the commercialization and development partner of choice in China because of our people, our global reach, and our relentless commitment to patients, to compliance and to quality," said John V. Oyler, Co-Founder, Chief Executive Officer, and Chairman of BeiGene. "During this past quarter we continued momentum for our planned upcoming product launches in the U.S. and China. As we look ahead to key catalysts over the rest of the year and into 2020, we’re on track for readouts from up to 10 ongoing Phase 3 or potentially registration-enabling studies in addition to the planned commercial launches."

Recent Business Highlights and Upcoming Milestones
Clinical Programs
Zanubrutinib, an investigational small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to maximize BTK occupancy and minimize off-target effects

Announced U.S. FDA acceptance and priority review of a new drug application (NDA) in patients with relapsed/refractory (R/R) mantle cell lymphoma (MCL) with a Prescription Drug User Fee Act (PDUFA) target action date of February 27, 2020; and

Initiated the following clinical trials:

A global Phase 3 clinical trial (NCT04002297) comparing zanubrutinib plus rituximab to bendamustine plus rituximab in patients with previously untreated MCL who are ineligible for stem cell transplant; and

A global Phase 2 clinical trial (NCT04116437) in patients with previously treated chronic lymphocytic leukemia (CLL) / small lymphocytic lymphoma (SLL) who are intolerant of prior treatment with ibrutinib.
Expected Milestones for Zanubrutinib

Present initial results from the 17p deletion arm of the Phase 3 SEQUOIA trial of zanubrutinib with bendamustine plus rituximab in previously untreated patients with CLL/SLL; updated results from a Phase 1/2 trial in R/R CLL/SLL; and updated results from the combination trial of zanubrutinib and tislelizumab in B-cell lymphoid malignancies at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) meeting (December 7-10, 2019, Orlando, FL);

Announce top-line results from the Phase 3 ASPEN trial comparing zanubrutinib to ibrutinib in patients with Waldenström’s Macroglobulinemia (WM) in 2019;

Receive U.S. FDA approval on the New Drug Application (NDA) in patients with R/R MCL, which has a PDUFA date of February 27, 2020;

Receive approvals in China for the treatment of patients with R/R MCL and R/R CLL/SLL in the first half of 2020;

File a supplemental new drug application (sNDA) in China for WM in the first half of 2020;

Announce top-line data from the SEQUOIA trial as early as 2020; and

Complete enrollment in the Phase 3 ALPINE trial comparing zanubrutinib with ibrutinib in patients with R/R CLL/SLL and in the Phase 2 MAGNOLIA trial in patients with R/R marginal zone lymphoma (MZL) in 2019 or early 2020.
Tislelizumab, an investigational humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages

Completed enrollment in the global Phase 3 clinical trial (NCT03412773) comparing tislelizumab to sorafenib in first-line patients with unresectable hepatocellular carcinoma (HCC);

Presented data from the Phase 2 clinical trial (NCT04004221) of tislelizumab in patients in China and South Korea with locally advanced or metastatic urothelial carcinoma (UC) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2019; and

Presented clinical data at the 22nd Annual Meeting of the Chinese Society of Clinical Oncology (CSCO), including:

Results from a Phase 2 trial (NCT03432598) of tislelizumab plus chemotherapy as first-line treatment for patients with lung cancer in China;

Updated results from a Phase 2 trial (NCT03469557) of tislelizumab plus chemotherapy in patients with esophageal squamous cell carcinoma (ESCC) in China; and

Results from a Phase 1/2 trial (CTR20160872) of tislelizumab in patients in China with advanced solid tumors.
Expected Milestones for Tislelizumab

Receive NDA approval in China for the treatment of patients with R/R classical Hodgkin’s lymphoma (cHL) in 2019;

Receive sNDA approval in China for the treatment of patients with locally advanced or metastatic UC in 2020;

Have regulatory discussions based on preliminary results from the global Phase 2 trial (NCT03419897) of tislelizumab in second- or third-line patients with HCC in 2019 or early 2020;

Announce top-line results from the Phase 3 trial (NCT03594747) comparing tislelizumab plus chemotherapy to chemotherapy alone in first-line patients with squamous non-small cell lung cancer (NSCLC) in China in 2020;

Announce top-line results from the Phase 3 trial (NCT03663205) comparing tislelizumab plus chemotherapy to chemotherapy alone in first-line patients with non-squamous NSCLC in China in 2020; and

Complete enrollment in the global portion of the second-line Phase 3 trial (NCT03358875) comparing tislelizumab with docetaxel in patients with NSCLC in 2019 or early 2020 and in the global Phase 3 trial (NCT03430843) comparing tislelizumab with chemotherapy in second-line patients with advanced ESCC in the first half of 2020.
Pamiparib, an investigational small molecule PARP inhibitor

Announced clinical data at ESMO (Free ESMO Whitepaper), including:
–
Updated results from the Phase 1b trial (NCT03150810) of pamiparib in combination with low-dose temozolomide in patients with locally advanced or metastatic solid tumors; and
–
Updated dose-escalation/expansion results from the Phase 1 trial (NCT02361723) of pamiparib in patients with advanced solid tumors.
Expected Milestones for Pamiparib

Have regulatory discussions based on preliminary results from the pivotal Phase 2 trial (NCT03333915) of pamiparib in Chinese patients with previously treated ovarian cancer (OC) in 2020;

Announce clinical data from the Phase 3 trial (NCT03519230) of pamiparib as a maintenance treatment in patients with platinum-sensitive recurrent OC in 2020;

Present data from the global Phase 1 trial (NCT02361723) of pamiparib in patients with OC and updated data from the Phase 1 trial (NCT02660034) of pamiparib in combination with tislelizumab in patients with advanced solid tumors in 2020; and

Submit NDA in China for OC in 2020.
Lifirafenib (BGB-283), an investigational RAF dimer inhibitor

Initiated a Phase 1b trial (NCT03905148) with SpringWorks Therapeutics of lifirafenib in combination with MEK inhibitor mirdametinib (formerly PD-0325901) in patients with advanced or refractory solid tumors.

BGB-A1217, an investigational TIGIT monoclonal antibody

Initiated patient enrollment in a Phase 1a/1b trial (NCT04047862) in China and Australia investigating the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of BGB-A1217 in combination with tislelizumab in patients with advanced solid tumors.
BGB-11417, an investigational small molecule Bcl-2 inhibitor

Completed preclinical and investigational new drug (IND) -enabling studies of BGB-11417, which demonstrated potent activity and high selectivity against the pro-apoptotic protein Bcl-2.
Expected Milestone for BGB-11417

Initiate Phase 1 studies in Australia and the United States to investigate the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of BGB-11417 in patients with mature B-cell malignancies in the first half of 2020.
Manufacturing Facilities

Completed the initial phase of construction and facility validation, and officially opened our biologics manufacturing facility in Guangzhou, China.
Commercial Operations

REVLIMID received formal inclusion on the national reimbursement drug list (NRDL) in China for R/R multiple myeloma;

In China, preparations for the planned launch of tislelizumab in patients with R/R cHL are ongoing, with field sales training now complete; and

In the U.S., the commercial field force has been hired in anticipation of the planned launch of zanubrutinib in patients with R/R MCL.
Corporate Developments

Announced a global strategic oncology collaboration with Amgen to commercialize XGEVA (denosumab), KYPROLIS (carfilzomib), and BLINCYTO (blinatumomab) in China and jointly develop 20 Amgen oncology pipeline assets. Amgen has agreed to purchase approximately $2.7 billion of BGNE shares. The transaction is expected to close in early 2020, subject to approval by a majority vote of the Company’s shareholders pursuant to the listing rules of the Hong Kong Stock Exchange, the expiration or termination of applicable waiting periods under applicable antitrust laws, and satisfaction of other customary closing conditions. Shareholders of the Company holding an aggregate of approximately 40% of the outstanding shares have agreed to vote in favor of the transactions; and

Announced a global license agreement with Seattle Genetics to in-license an advanced preclinical oncology candidate in Asia (except Japan) and the rest of the world other than the Americas (United States, Canada and Latin American countries) and Europe. The agent is expected to advance into clinical trials in the first half of 2020.
Third Quarter 2019 Financial Results
Cash, Cash Equivalents, Restricted Cash and Short-Term Investments were $1.28 billion as of September 30, 2019, compared to $1.56 billion as of June 30, 2019 and $1.81 billion as of December 31, 2018.

Cash used by operating activities for the three months ended September 30, 2019 was $265.01 million, compared to $132.19 million for the same period in 2018. The increase in the use of cash was primarily attributable to the continued ramp-up in operating expenses in support of our preparation for commercial launch of our late-stage drug candidates in the U.S. and China, continued development of our internal and in-licensed drug candidates, as well as overall organizational growth.

Capital expenditures were $30.87 million for the three months ended September 30, 2019, which related primarily to the construction of our Guangzhou biologics facility.

Revenue for the quarter ended September 30, 2019 was $50.14 million, compared to $54.20 million in the same period in 2018. The decrease is primarily attributable to the termination of the Celgene collaboration agreement for tislelizumab in the second quarter of 2019, and as a result, the cessation of any related collaboration revenue.

Product revenue from sales of ABRAXANE, REVLIMID and VIDAZA in China totaled $50.14 million for the quarter ended September 30, 2019, compared to $38.45 million for the quarter ended September 30, 2018. Sales in the third quarter of 2019 were negatively impacted by temporary supply disruptions of ABRAXANE.

Collaboration revenue was nil for the quarter ended September 30, 2019, compared to $15.76 million for the same period in 2018. The decrease is attributable to the termination of the Celgene collaboration agreement on tislelizumab in the second quarter of 2019.
Expenses for the quarter ended September 30, 2019 were $362.41 million, compared to $205.30 million in the same period in 2018.

Cost of sales for the quarter ended September 30, 2019 were $20.11 million, compared to $8.71 million in the same period in 2018. Cost of sales includes the acquisition costs related to the amount of ABRAXANE, REVLIMID and VIDAZA that was sold during the period in China. Costs to manufacture inventory in preparation for commercial launch, incurred prior to regulatory approval, are charged to research and development expense as incurred.

R&D Expenses for the quarter ended September 30, 2019 were $236.97 million, compared to $147.59 million in the same period in 2018. 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, preparation for regulatory submissions, and manufacturing costs related to pre-commercial activities and supply. Employee share-based compensation expense also contributed to the overall increase in R&D expenses, and was $20.67 million for the quarter ended September 30, 2019, compared to $15.52 million for the same period in 2018, due to increased headcount.

SG&A Expenses for the quarter ended September 30, 2019 were $105.00 million, compared to $48.82 million in the same period in 2018. 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 commercial products in China and the planned launches of our late-stage drug candidates in the U.S. and China, as well as higher professional service fees and costs to support our growing operations. The overall increase in SG&A expenses was also attributable to higher SG&A-related share-based compensation expense, which was $16.14 million for the quarter ended September 30, 2019, compared to $9.61 million for the same period in 2018, due to increased headcount.

Net Loss for the quarter ended September 30, 2019 was $307.36 million, or $0.39 per share, or $5.11 per American Depositary Share (ADS), compared to $144.03 million, or $0.19 per share, or $2.53 per ADS in the same period in 2018.

On November 12, 2019 Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported financial results for the third quarter ended September 30, 2019 (Press release, TapImmune, NOV 12, 2019, View Source [SID1234550981]).

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"We continue to make progress in advancing our next-generation T-cell based immunotherapies for the treatment of hematological malignancies and solid tumors," said Peter L. Hoang, President and CEO of Marker Therapeutics. "Our partner-sponsored MultiTAA T-cell therapy trials at the Baylor College of Medicine continue to show promising results. In addition, we continue to expand our team and build out our infrastructure to support future Marker-sponsored clinical trials. We expect the next 12 to 18 months to be an exciting and productive time for our Company."

Continued Mr. Hoang: "We recently filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for our MultiTAA T-cell therapy as part of a planned Marker Phase 2 study in post-allogeneic hematopoietic stem cell transplant patients with acute myeloid leukemia in both the adjuvant and active disease setting. The FDA reviewed our submission and requested additional information regarding certain quality and technical specifications for two reagents supplied by third party vendors that are used in our manufacturing process. Because the FDA requires these data in order to clear the IND, the Marker AML trial has been placed on clinical hold until our complete response to the technical questions is satisfactory to the FDA. While these reagents are not present in the final product, we worked with respective manufacturers of these reagents to satisfy the FDA’s questions and subsequently submitted a complete response to the FDA in late October. We currently project to initiate our Phase 2 trial in 2020 and look forward to providing an update on our clinical path forward upon receiving the FDA’s feedback."

PROGRAM UPDATES

Multi-Antigen Targeted (MultiTAA) T-Cell Therapies

Marker Submits Response to FDA Clinical Hold on AML Trial

The Company worked with regulatory and quality groups at the respective manufacturers to address the FDA’s request and submitted a complete response to the issues raised by the FDA on October 28, 2019. The FDA will respond within 30 days after receiving Marker’s complete response, indicating whether the hold is lifted and, if not, specifying the reasons the clinical trial remains on hold. Marker expects to initiate its Phase 2 clinical trial of MultiTAA therapy for the treatment of post-transplant AML in 2020.

T Cell-Based Vaccines

Phase 2 Triple Negative Breast Cancer Trial Progressing

Marker continues to advance its T cell-based vaccine program in triple negative breast cancer. To date, results have shown:

·Based on a preliminary analysis of 34 patients enrolled in the triple negative breast cancer trial, 31 patients showed meaningful immune response to vaccine treatment;
·Of 80 patients treated at 11 clinical sites, 14 have shown disease progression, as of September 30, 2019, following treatment with TPIV200.

Phase 2 Platinum-Sensitive Advanced Ovarian Cancer Trial Update

Marker will be discontinuing the development of TPIV200 in patients with platinum-sensitive advanced ovarian cancer based on an unblinded review of interim results from its Phase 2 study conducted by an independent Data and Safety Monitoring Board (DSMB). Although the DSMB did not express any safety concerns with respect to TPIV200, Marker has elected to suspend the trial because it did not meet the threshold for probability of success based upon the Company’s pre-specified criteria. Pending full review of the data, Marker anticipates closing the trial in the first quarter of 2020.

CORPORATE UPDATES

·Nadia Agopyan, Ph.D., RAC, former Director of Regulatory Affairs, Global Regulatory Lead at Kite Pharma, appointed as Vice President of Regulatory Affairs
·Steve Elms, Managing Partner at Aisling Capital, appointed to Board of Directors

THIRD QUARTER 2019 FINANCIAL RESULTS

Net loss for the quarter ended September 30, 2019 was $5.5 million, compared to a net loss of $4.4 million for the quarter ended September 30, 2018.

Research and development expenses during the three months ended September 30, 2019 were $3.1 million, compared to $1.9 million during the three months ended September 30, 2018. The increase of $1.2 million was primarily attributable to increases in personnel-related expenses, relating to the build-up of Marker’s internal infrastructure.

General and administrative expenses were $2.5 million during the three months ended September 30, 2019 as compared to $2.6 million during the three months ended September 30, 2018. The decrease was primarily attributable to $0.6 million of merger-related expenses incurred during the three months ended September 30, 2018, offset by increased expenses in headcount-related and legal and other professional expenses.

CASH POSITION AND GUIDANCE

At September 30, 2019, Marker had cash and cash equivalents of $48.5 million. The Company believes that its existing cash and cash equivalents will fund its current operations through at least the fourth quarter of 2020.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its third quarter 2019 financial results and provide an update on recent corporate activities today at 5:00 p.m. EST.

The webcast will be accessible in the Investors section of the Company’s website at www.markertherapeutics.com. Individuals can participate in the conference call by dialing 877-407-8913 (domestic) or 201-689-8201 (international) and referring to the "Marker Therapeutics Third Quarter 2019 Earnings Call."

The archived webcast will be available for replay on the Marker website following the event.

On November 12, 2019 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical stage biotechnology company focused on discovering and developing novel small molecule drugs for the treatment of cancer and other life-threatening diseases, reported its financial results for the third quarter ended September 30, 2019 (Press release, Calithera Biosciences, NOV 12, 2019, View Source [SID1234550980]). As of September 30, 2019, cash equivalents and investments totaled $133.6 million.

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"This was a productive quarter for Calithera, during which we achieved multiple, key milestones in our clinical and pipeline programs," said Susan Molineaux, PhD, president and chief executive officer of Calithera. "This includes completing enrollment in our registrational CANTATA trial evaluating telaglenastat for the treatment of patients with renal cell carcinoma, and presenting new data on several programs at both the ESMO (Free ESMO Whitepaper) and SITC (Free SITC Whitepaper) annual meetings. In addition, we successfully completed a Phase 1 trial in healthy volunteers of CB-280, an oral arginase inhibitor for the treatment of cystic fibrosis."

Third Quarter 2019 and Recent Highlights

Completed patient enrollment of randomized CANTATA trial of telaglenastat with cabozantinib in advanced renal cell carcinoma. The CANATA trial is a global, randomized, double-blind clinical trial of telaglenastat combined with cabozantinib, in patients with advanced or metastatic renal cell carcinoma who have received one or two prior treatments. The CANTATA trial enrolled 445 patients at multiple centers globally. The primary endpoint is progression-free survival. Calithera plans to report top-line efficacy and safety data from the trial in the second half of 2020.

Presented results of Phase 2 ENTRATA study of telaglenastat (CB-839) with everolimus in renal cell carcinoma at the ESMO (Free ESMO Whitepaper) 2019 Congress. The ENTRATA trial (NCT03163667) was a Phase 2 randomized, double-blind trial designed to evaluate the safety and efficacy of telaglenastat in combination with everolimus versus placebo with everolimus in patients with advanced clear cell RCC who have been treated with at least two prior lines of systemic therapy, including at least one prior VEGFR-targeted tyrosine kinase inhibitor. The trial enrolled 69 patients at multiple centers in the United States. The primary endpoint of ENTRATA was progression-free survival (PFS). Telaglenastat, when added to everolimus, doubled the median PFS in heavily pretreated patients with advanced RCC to 3.8 months as compared to 1.9 months for everolimus alone, and reduced the risk of disease progression or death by 36% (HR=0.64, p=0.079 one-sided). The primary endpoint of the trial was PFS per investigator assessment with a predetermined threshold of p£0.2 one-sided. The secondary endpoint of overall survival is not yet mature.

Initiated Phase 1/2 clinical trial of telaglenastat in combination with palbociclib for solid tumors. The Phase 1/2 clinical trial is evaluating telaglenastat in combination with Pfizer’s CDK4/6 inhibitor palbociclib, also known as Ibrance. The study will evaluate the safety and anti-tumor activity of telaglenastat plus palbociclib in patients with KRAS-mutated colorectal cancer (CRC) and KRAS-mutated non-small cell lung cancer (NSCLC).

Presented new INCB001158 data at the ESMO (Free ESMO Whitepaper) 2019 Congress. Calithera and Incyte are collaborating to conduct this Phase 1 study evaluating INCB001158 as monotherapy and in combination with the PD-1 inhibitor pembrolizumab in checkpoint inhibitor refractory and naïve advanced/metastatic solid tumors. Responses were observed in patients with microsatellite stable (MSS) colorectal cancer, a disease not historically sensitive to checkpoint inhibition.

Completed a Phase 1 clinical trial of CB-280 in healthy volunteers. The first-in-human Phase 1 trial evaluated the safety, tolerability and pharmacokinetic profile of oral CB-280 in healthy volunteers. A phase 1b clinical study in cystic fibrosis patients is expected to start enrollment in the first half of 2020.

Presented new preclinical data for IL4I1 and CD73 programs at the SITC (Free SITC Whitepaper) Annual Meeting. CB-708 is a selective, oral inhibitor of CD73, an enzyme that synthesizes the immunosuppressive agent adenosine and is over-expressed in multiple tumor types. By blocking adenosine production in the tumor, CB-708 is designed to enhance T-cell activation, leading to anti-tumor activity. Interleukin 4 (IL-4)-Induced Gene 1 (IL4I1) is an enzyme that is primarily expressed by tumor cells and antigen presenting cells, and produces hydrogen peroxide, an inhibitor of T-cell function. IL4I1 has a potential role in immune evasion, and inhibition may enhance an effective anti-tumor immune response. Calithera announced the IL4I1 inhibitor program this quarter.

Selected Third Quarter 2019 Financial Results

Cash, cash equivalents and investments totaled $133.6 million at September 30, 2019.

Research and development expenses were $17.2 million for the three months ended September 30, 2019, compared with $16.4 million for the same period in the prior year. The increase of $0.8 million was primarily due to a $0.7 million increase in the telaglenastat program, including for the CANTATA trial, an increase of $0.4 million in the INCB001158 program, and an increase of $0.5 million in our early-stage research programs, partially offset by a decrease of $0.8 million in our CB-280 program.

General and administrative expenses were $3.9 million for the three months ended September 30, 2019, compared with $3.1 million for the same period in the prior year. The increase of $0.8 million primarily related to higher professional services costs and personnel-related costs.

Net loss for the three months ended September 30, 2019 was $20.3 million, or $0.38 per share.

Conference Call Information

Calithera will host an update conference call today, Tuesday, November 12, at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time. The call may be accessed by dialing (855) 783-2599 (domestic) or (631) 485-4877 and referring to conference ID 6499000. To access the live audio webcast or the subsequent archived recording, visit the Investors section of the Calithera website at www.calithera.com. The webcast will be recorded and available for replay on Calithera’s website for 30 days.