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.

On November 12, 2019 Adaptive Biotechnologies Corporation ("Adaptive Biotechnologies") (Nasdaq: ADPT) reported financial results for the quarter ended September 30, 2019 (Press release, Adaptive Biotechnologies, NOV 12, 2019, View Source [SID1234550979]).

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"During the third quarter, we continued to make strong progress across our existing products and our pipeline," said Chad Robins, chief executive officer and co-founder of Adaptive. "We are encouraged to see increasing demand for clonoSEQ as we continue developing the market and expanding our label. We were also excited to confirm our first clinical signal for immunoSEQ Dx, proving that we can read and translate how the adaptive immune system diagnoses disease."

Recent Highlights

Revenue of $26.1 million for the third quarter of 2019, an increase of 52% over the third quarter of 2018

Achieved milestone for immunoSEQ Dx with confirmation of the first clinical signal in acute Lyme disease

Entered into a partnership for use of the clonoSEQ Assay as preferred MRD test for drug development across Amgen’s hematology franchise

Entered into a partnership with Illumina to enable the development of IVD test kits for our diagnostic products

Third Quarter 2019 Financial Results

Revenue was $26.1 million for the quarter ended September 30, 2019, representing a 52% increase from the third quarter in the prior year. Sequencing revenue was $11.7 million for the quarter, representing a 38% increase from the third quarter in the prior year. Development revenue increased to $14.4 million for the quarter, representing a 65% increase from the third quarter in the prior year.

Operating expenses were $44.1 million for the third quarter of 2019, compared to $26.3 million in the third quarter of the prior year, representing an increase of 68%.

Net loss was $14.0 million for the third quarter of 2019, compared to $8.3 million for the same period in 2018.

Adjusted EBITDA (non-GAAP) was a loss of $12.7 million for the third quarter of 2019, compared to a loss of $5.1 million for the third quarter of the prior year.

Cash, cash equivalents and marketable securities was $708.7 million as of September 30, 2019.

2019 Financial Guidance

Management will provide updates to the 2019 revenue outlook on the conference call scheduled to discuss the third quarter 2019 financial results.

Webcast and Conference Call Information

Adaptive Biotechnologies will host a conference call to discuss its third quarter 2019 financial results after market close on Tuesday, November 12, 2019 at 4:30 PM Eastern Time. The conference call can be accessed live over the phone (800) 361-2311 for U.S. callers or (409) 937-8761 for international callers (Conference ID: 3698520). The webcast can be accessed at View Source

On November 12, 2019 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the third quarter ended September 30, 2019 and provided a summary of recent events (Press release, Regulus, NOV 12, 2019, View Source [SID1234550978]).

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"We recently received the final reports from the chronic toxicity studies in mice and non-human primates and are preparing our submission of our complete response to Food and Drug Administration ("FDA") in our efforts to address the requirements to reinitiate the Multiple Ascending Dose ("MAD") clinical study for RGLS4326," said Jay Hagan, CEO of Regulus. "We look forward to FDA’s feedback and continued productive dialogue."

Third Quarter 2019 Corporate Highlights and Recent Updates

Management Transition to New Chief Financial Officer: In July 2019, the Company appointed Cris Calsada as its new Chief Financial Officer, effective August 30, 2019. Ms. Calsada’s appointment follows the resignation of the Company’s previous Chief Financial Officer, Dan Chevallard, in July 2019.

New Lease Agreement Significantly Reduces Contractual Lease Obligations: In June 2019, the Company entered into an amendment of its lease (the "Lease Amendment") of 24,562 square feet located at 10628 Science Center Drive Suite 100, San Diego, California 92121. Under the terms of the Lease Amendment, the expiration of the lease was accelerated from June 30, 2023 to June 30, 2019, and the lease terminated on July 1, 2019. Concurrently with the Lease Amendment, the Company entered into a new lease agreement for 8,727 square feet located at 10628 Science Center Drive, Suite 225, San Diego, California, 92121, which it uses as its new principal offices and laboratory for research and development. This relocation reduced the Company’s facility size by approximately 65% and reduced its future contractual lease obligations by approximately 78%.

Program Updates

RGLS4326 for Autosomal Dominant Polycystic Kidney Disease ("ADPKD"): RGLS4326, a novel oligonucleotide designed to inhibit miR-17, is in Phase 1 clinical development for the treatment of ADPKD and currently on a partial clinical hold. In January 2019, the Company submitted a comprehensive data package for RGLS4326 to FDA that included the results from the planned 13-week interim analysis of the repeat mouse chronic toxicity study, as well as results from additional investigations, analytical testing, additional data from the previously terminated mouse chronic toxicity study, data from the completed Phase I single ascending dose ("SAD") study and data from the first cohort of the Phase I MAD study, to support its plan to resume the Phase I MAD study. After review of the requested submission,

FDA notified the Company in July 2019 of additional nonclinical data requirements and placed the IND on a partial clinical hold, formalizing the specific requirements to initiate the MAD study and further proceed into chronic dosing in ADPKD patients. The additional data requirements have been outlined in two parts. In order to resume the MAD study, FDA has requested the final reports from the chronic toxicity studies in both mice and non-human primates and satisfactory related analyses to ensure subjects can be safely dosed. Additional information and analyses from new nonclinical studies, planned to be generated over the next several quarters, are required for chronic dosing and will be submitted at a later date. The Company recently received the final reports from the chronic toxicity studies in mice and non-human primates and is preparing to submit a complete response to the partial clinical hold in order to be able to resume the MAD study, pending agreement with FDA. Regulus anticipates a response from FDA before the end of the year. Regulus is allowed to proceed with additional SAD clinical studies as part of the process to gather additional supporting information to guide the future development of the program.

In September 2019, the Company announced that Nature Communications published an article entitled, "Discovery and preclinical evaluation of anti-miR-17 for the treatment of polycystic kidney disease." The article highlights preclinical research that identifies RGLS4326, a first-in-class anti-miR-17 oligonucleotide, as a potential disease-modifying treatment for ADPKD. In addition, a poster highlighting this work was presented at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting held October 13-16, 2019 in Munich, Germany.

Third Quarter 2019 Financial Results

Cash Position: As of September 30, 2019, Regulus had $14.6 million in cash and cash equivalents.

Revenue: Revenue was less than $0.1 million and $6.8 million for the three and nine months ended September 30, 2019, respectively, compared to less than $0.1 million and $0.1 million for the three and nine months ended September 30, 2018. The increase for the nine months ended September 30, 2019 was attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of RG-012.

Research and Development (R&D) Expenses: R&D expenses were $2.4 million and $10.3 million for the three and nine months ended September 30, 2019, compared to $6.9 million and $28.7 million for the same periods in 2018. The decreases were driven by decreases in external development expenses, primarily attributable to the voluntary pause of the RGLS4326 Phase 1 MAD clinical study in the third quarter of 2018 and commencement of the transfer of the RG-012 program to Sanofi in the fourth quarter of 2018. Additionally, the decreases were driven by reductions in personnel and internal expenses, primarily attributable to a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.

General and Administrative (G&A) Expenses: G&A expenses were $2.6 million and $9.0 million for the three and nine months ended September 30, 2019, compared to $3.0 million and $10.1 million for the same periods in 2018. These amounts reflect personnel-related and ongoing general business operating costs. The decreases were driven by a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.

Net Loss: Net loss was $5.4 million, or $0.26 per share (basic and diluted), and $13.7 million, or $0.86 per share (basic and diluted), for the three and nine months ended September 30, 2019, respectively, compared to $10.3 million, or $1.18 per share (basic and diluted), and $40.1 million, or $4.62 per share (basic and diluted), for the same periods in 2018. Historical and current period net loss per share values have been retroactively adjusted to reflect our October 2018 reverse stock split.

About Autosomal Dominant Polycystic Kidney Disease (ADPKD)

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a partial clinical hold by the U.S. Food and Drug Administration.