On November 4, 2019 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results for the third quarter ended September 30, 2019 and provided a general update on clinical and corporate developments (Press release, Deciphera Pharmaceuticals, NOV 4, 2019, View Source [SID1234550231]).

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"We believe the Breakthrough Therapy Designation we received from the FDA underscores the potential for ripretinib to transform the treatment landscape for patients with advanced GIST," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "Based on the positive results from the Phase 3 INVICTUS study, we remain on track for our planned NDA submission for ripretinib in the first quarter of 2020. In addition, we provided important new clinical updates on rebastinib and DCC-3014 at the recent AACR (Free AACR Whitepaper)-NCI-EORTC meeting and we continue to progress our broad portfolio of novel product candidates all discovered here at Deciphera."

Recent Highlights and Upcoming Milestones

Ripretinib
Presented positive top-line data from the INVICTUS pivotal Phase 3 clinical study evaluating the safety and efficacy of ripretinib in fourth-line and fourth-line plus GIST patients.
FDA granted Breakthrough Therapy Designation (BTD) for ripretinib for the treatment of patients with advanced GIST who have received prior treatment with imatinib, sunitinib and regorafenib.
Company expects to submit a New Drug Application (NDA) to the FDA for ripretinib for the treatment of patients with advanced GIST who have received prior treatment with imatinib, sunitinib and regorafenib in the first quarter of 2020.
Presented updated data from the ongoing Phase 1 clinical study of ripretinib in patients with second-line through fourth-line plus GIST at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). The Company believes the updated data continue to support the ongoing INTRIGUE pivotal Phase 3 clinical study comparing ripretinib to sunitinib for the treatment of second-line GIST patients who have previously received imatinib.
Established ripretinib Expanded Access Program (EAP) for patients with locally advanced unresectable or metastatic GIST who have received treatment with prior therapies. The ripretinib EAP provides a pathway for eligible patients to gain access to this investigational medicine outside of clinical trials when no comparable or satisfactory alternative therapy option is available.
DCC-3014
Presented data from the Phase 1 dose escalation study of DCC-3014, an oral inhibitor of CSF1R, in patients with advanced solid tumors, at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). The Phase 1 data demonstrated tolerability, pharmacokinetics and biomarker mechanistic proof-of-concept in patients with advanced malignancies.
Company plans to present preliminary data from initial tenosynovial giant cell tumor (TGCT) patients at the 2019 Connective Tissue Oncology Society (CTOS) Annual Meeting being held November 13-16 in Tokyo, Japan.
Rebastinib
Presented data from the ongoing Phase 1b/2 clinical study of rebastinib, an oral TIE2 kinase inhibitor, in combination with paclitaxel at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). In Part 1 of the ongoing Phase 1b/2 study, the combination of rebastinib and paclitaxel exhibited encouraging preliminary anti-tumor activity across treatment arms and was generally well tolerated.
DCC-3116
Presented preclinical data for DCC-3116, a potential first-in-class autophagy inhibitor designed to treat mutant RAS cancers at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). In vivo and in vitro data demonstrated DCC-3116 is a potent, selective and tight-binding inhibitor of ULK kinase, and represents a differentiated approach to autophagy inhibition and a potential first-in-class opportunity for a new therapeutic modality in mutant RAS cancers.
Recent Corporate Updates

In October 2019, Deciphera announced the appointment of Matthew L. Sherman, M.D. as Executive Vice President and Chief Medical Officer. Dr. Sherman brings over 25 years of experience as a physician-scientist in clinical drug development in oncology and hematology at leading biotechnology and pharmaceutical companies.
In the third quarter of 2019, Deciphera announced the closing of an underwritten public offering of 12,432,431 shares at a public offering price of $37.00 per share, which included the exercise in full by the underwriters of their option to purchase up to 1,621,621 shares of common stock. Total net proceeds to Deciphera were approximately $431.8 million, after deducting underwriting discounts and commissions and other offering expenses.
Third Quarter 2019 Financial Results

Cash Position: As of September 30, 2019, cash, cash equivalents and marketable securities were $634.6 million, compared to cash and cash equivalents of $293.8 million as of December 31, 2018. Deciphera expects its cash, cash equivalents and marketable securities as of September 30, 2019 will enable the Company to fund its operating expenses, capital expenditure requirements and debt service payments into 2022.
R&D Expenses: Research and development expenses for the third quarter of 2019 were $40.4 million, compared to $20.6 million for the same period in 2018. The increase was primarily due to the Company’s clinical trial costs related to the INTRIGUE pivotal Phase 3 study in second-line GIST. Personnel-related costs increased to $11.6 million primarily due to an increase in headcount and stock-based compensation expense in the research and development function. Personnel-related costs for the third quarters of 2019 and 2018 included non-cash, stock-based compensation expense of $2.0 million and $1.1 million, respectively. Facility-related and other costs included increased consultant fees of $0.4 million and increased costs incurred in connection with our early-stage drug discovery programs of $0.2 million.
G&A Expenses: General and administrative expenses for the third quarter of 2019 were $18.0 million, compared to $5.3 million for the same period in 2018. The increase was primarily due to increases in headcount and stock-based compensation expense in the Company’s general and administrative function. Non-cash, stock-based compensation was $2.7 million and $1.5 million for the third quarters of 2019 and 2018, respectively.
Net Loss: For the third quarter of 2019, Deciphera reported a net loss of $56.2 million, or $1.28 per share, compared with a net loss of $24.4 million, or $0.65 per share, for the same period in 2018.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, November 4, 2019, at 4:30 PM ET. To access the live call by phone please dial (866) 930-5479 (domestic) or (409) 216-0603 (international); the conference ID is 1181263. A live audio webcast of the event may also be accessed through the "Investors" section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On November 4, 2019 CTI BioPharma Corp. (Nasdaq: CTIC) reported its financial results for the third quarter and nine months ended September 30, 2019 (Press release, CTI BioPharma, NOV 4, 2019, View Source [SID1234550230]).

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"We advanced our pacritinib development program in the third quarter, and recently took an important step forward for the company by initiating and enrolling the first patient in the PACIFICA trial, our pivotal Phase 3 trial of pacritinib in myelofibrosis patients with severe thrombocytopenia," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "An estimated one-third of patients with myelofibrosis are severely thrombocytopenic – a population with limited therapeutic options and poor survival, thereby making this disease setting a very important area of unmet medical need. In the near-term, we look forward to presenting results from the PAC203 Phase 2 trial at a scientific conference before the end of the year."

Third Quarter Financial Results
Operating loss was $9.7 million and $31.2 million for the three and nine months ended September 30, 2019, respectively, compared to operating loss of $14.8 million and $33.1 million for the respective periods in 2018. The decrease in operating loss during the three-month period ended September 30, 2019 as compared to the comparable period in 2018 resulted primarily from a decrease in operating expenses as well as the increase in license and contract revenues as discussed below. The decrease in operating loss for the nine months ended September 30, 2019 as compared to the same period in 2018 resulted primarily from a decrease in operating expenses, offset by the decrease in license and contract revenue between periods. As of September 30, 2019, cash, cash equivalents and short-term investments totaled $46.7 million, compared to $67.0 million as of December 31, 2018. CTI BioPharma expects current cash, cash equivalents and short-term investments will enable it to fund its operations into the third quarter of 2020.

License and contract revenues for the three and nine months ended September 30, 2019 were $2.3 million and $3.3 million, respectively, compared to $0.7 million and $12.2 million for the respective periods in 2018. The increase in license and contract revenues for the three months ended September 30, 2019 compared to the comparable period in 2018 is primarily due to revenue recognized in connection with the asset purchase agreement with our partner Les Laboratoires Servier and Institut de Recherches Internationales Servier. The decrease in license and contract revenues for the nine months ended September 30, 2019 compared to the same period in 2018 is primarily due to the recognition of $10.0 million in milestone revenue in 2018 from Teva Pharmaceutical Industries Ltd. related to the achievement of a milestone for FDA approval of TRISENOX (arsenic trioxide) for first-line treatment of acute promyelocytic leukemia. There were no such revenues for the comparable period in 2019.

Net loss attributable to common stockholders for the three months ended September 30, 2019 was $10.0 million, or $(0.17) for basic and diluted loss per share, compared to net loss attributable to common stockholders of $14.8 million, or $(0.26) for basic and diluted loss per share, for the same period in 2018. Net loss attributable to common stockholders for the nine months ended September 30, 2019 was $31.8 million, or $(0.55) for basic and diluted loss per share, compared to net loss attributable to common stockholders of $30.2 million, or $(0.55) for basic and diluted loss per share, for the same period in 2018.

On November 4, 2019 Ascentage Pharma (6855.HK), a globally-focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, hepatitis B virus and age-related diseases, reported a combination therapy strategic collaboration with Henlius (2696.HK), working together to conduct clinical trials of the combination therapy between APG-2575, a novel, orally administered Bcl-2 selective inhibitor developed by Ascentage Pharma, and HLX01 (Rituximab Injection), the first launched product by Henlius, for the treatment of chronic lymphocytic leukemia (CLL) in the People’s Republic of China (Press release, Ascentage Pharma, NOV 4, 2019, View Source [SID1234550229]).

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APG-2575 is a novel, orally administered Bcl-2 selective inhibitor developed by Ascentage Pharma. It is designed to treat hematologic malignancies by selectively blocking Bcl-2 to restore the normal apoptosis process in cancer cells. In July 2019, the first patient was dosed successfully in a Phase I clinical trial of APG-2575 for the treatment of hematologic malignancies in China, making APG-2575 potentially the first China-made Bcl-2 inhibitor to enter clinical study. CLL and non-Hodgkin’s lymphoma (NHL) patients are included in this trial. The Phase I clinical trial of APG-2575 in hematologic malignancies has already been initiated in Australia and the U.S. As of Aug. 13, 2019, one patient’s tumor had been reduced in size by over 60%, a response that met the criteria for partial response (PR); there were three patients in the 400mg dose cohort whose absolute lymphocyte counts (ALC), another key efficacy parameter of this study, have reached the criteria for complete response (CR) by the end of cycle 1. No tumor lysis syndrome (TLS) was observed in the study, indicating the favorable safety profile of the investigational agent. Moreover, at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, Ascentage Pharma has already presented results from several preclinical studies that demonstrated APG-2575’s potential in combination therapies.

As the first approved biosimilar in China, HLX01 (Rituximab Injection) is mainly for the treatment of non-Hodgkin’s lymphoma (NHL). In February 2019, China National Medical Products Administration (NMPA) approved HLX01 (Rituximab Injection) for the treatment of adult patients with 1) relapsed or refractory, follicular lymphoma; 2) previously untreated stages III-IV follicular, non-Hodgkin’s lymphoma; and 3) CD20-positive, diffuse large B-cell, non-Hodgkin’s lymphoma (DLBCL), namely all the approved indications of the originator Rituximab in China. Rituximab in combination with chemotherapy has long been the standard treatment of NHL. HLX01 (Rituximab Injection) provides an alternative treatment option for lymphoma patients, as over 1,000 patients benefited in the first month after its commercial launch.

The collaboration is of great significance to both companies. Bcl2 inhibitors are a rising star for B cell malignancies and can be applied to the treatment of CLL in combination with rituximab. Potentially this combination could also be extended to other B cell malignancies

Dr. Dajun Yang, Chairman and CEO of Ascentage Pharma, commented: "We are delighted to be entering this collaboration agreement with Henlius. As the first China-made Bcl-2 inhibitor to enter clinical study, APG-2575 is a key candidate in our development pipeline of apoptosis with a great potential in the treatment of hematologic malignancies. HLX01 (Rituximab Injection), as the first approved biosimilar in China, provides a new treatment option for lymphoma patients. Combination therapy is going to be the trend in future. The teams from Ascentage Pharma and Henlius will work closely to explore the clinical utility of this combination therapy. We hope APG-2575 combined with HLX01 will demonstrate synergistic effect in the treatment of CLL, and thereby offer additional treatment options for Chinese patients."

"We are excited to reach the agreement with Ascentage Pharma," said Scott Liu, Ph.D., Co-founder, President and CEO of Henlius. "As the first approved and marketed product of Henlius, HLX01 (Rituximab Injection) is approved for all the indications of the originator rituximab in China, thus providing alternative options for lymphoma patients. APG-2575 is indicated for multiple hematologic malignancies as mono-therapy and shows great potential in combination therapy. We expect the two products to join hand in hand in the treatment of CLL. In future, Henlius and Ascentage Pharma will press forward with the combination therapy, and provide more treatment options for patients in China."

On November 4, 2019 Harbour BioMed (HBM) reported the start of the first clinical trial of its next-generation fully human anti-CTLA-4 antibody (HBM4003) for the treatment of patients with advanced solid tumors (Press release, Harbour BioMed, NOV 4, 2019, View Source [SID1234550228]). This trial is the first with a fully human antibody based on heavy chain only antibody technology (HCAb). HBM4003 has shown potential for increased anti-tumor activity based on enhanced antibody dependent cell toxicity (ADCC) mediated Treg depletion and a favorable safety profile resulting from reduced half-life.

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In preclinical studies, HBM4003 demonstrated potent anti-tumor activity with much lower systemic drug exposure, suggesting a potentially significant improvement in its therapeutic profile. HBM presented preclinical study results of HBM4003 in April 2019 at the annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper). The trial is design led to assess the safety, pharmacokinetic profile and preliminary anti-tumor activity of HBM4003 in patients with advanced solid tumors.

"HBM4003, based on our proprietary human HCAb platform, underscores the potential for heavy chain only antibodies in developing the next-generation of immuno-oncology therapeutics for patients," said Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed. "This study, being conducted in Australia, is the first part of a global development program, with US and China clinical trials — including combination studies — expected to begin in the near future." Dr. Wang added, "HBM is rapidly building its pipeline, with HBM4003 advancing to the clinic in less than three years and with additional mono- and bi-specific antibodies against various targets in pre-clinical development."

CTLA-4 is one of the major negative regulators of T-cell responses. Anti-CTLA-4 antibodies are immune checkpoint inhibitors with proven value in cancer immunotherapy by increasing T-cell activity to attack tumor cells. Despite demonstrated efficacy, their safety profiles have been a barrier to broader application as both mono- and combination therapies. Preclinical studies have shown that HBM4003’s immune stimulatory activity is driven by two mechanisms: depletion of immune suppressing Treg through enhanced ADCC and; inhibition of negative signaling from the interaction of CTLA-4 with the co-stimulatory molecule B7.

"Advanced solid tumors including melanoma remain a major challenge despite improvements in the standard of care," said Prof. Paul de Souza, Dean of Medicine at the University of Wollongong Australia, and a principal investigator for this study. "Preclinical studies of HBM4003 have shown great potential. This trial provides the first opportunity to evaluate how this promising compound will perform in the treatment of solid tumors."

About Heavy Chain Only Antibodies (HCAb)

Heavy-chain only antibodies consist only of two heavy chains; they lack the two light chains usually found in traditional antibodies. HCAb’s are smaller than conventional antibodies, potentially allowing for increased tissue penetration. These antibodies also maintain IgG-like pharmacokinetic properties and immune activation (Fc) functions. HCAbs can bind antigens despite having only VH domains and have shown similar specificity to regular antibodies.

HBM’s HCAbs, which are generated by its fully human transgenic mouse platform, further enable the design of versatile formats of antibodies capable of addressing different mechanisms of action and use in different applications. HCAbs are naturally generated human antibodies against desired targets; thus there is no need for humanization to reduce immunogenicity.

On November 4, 2019 ChemoCentryx, Inc., (Nasdaq:CCXI), reported financial results for the third quarter ended September 30, 2019 and provided an overview of the Company’s recent corporate highlights (Press release, ChemoCentryx, NOV 4, 2019, View Source [SID1234550227]).

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"Strong and swift is the pace of our progress as we eagerly anticipate topline data from our Phase III pivotal ADVOCATE trial of avacopan in ANCA vasculitis," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "At our recent R&D Day in New York the stage was set for the upcoming data, as our schedule featured two pre-eminent nephrologists as well as an ANCA vasculitis patient who highlighted the patient journey, the disease burden and the noxious consequences of chronic steroid use as part of the current standard-of-care. We provided a comprehensive overview of ANCA disease assessment and therapeutic improvement—ranging from the primary endpoints, BVAS remission at 26 and 52 weeks, to key secondary endpoints in the trial, as we aim to demonstrate a new way to treat this deadly disease."

"Furthermore, topline data from ADVOCATE is just the first in a cadence of five data readouts expected between now and the end of 2020. These readouts include those from the LUMINA-1 and LUMINA-2 trials of our CCR2 inhibitor, CCX140, in Focal Segmental Glomerulosclerosis (FSGS), the ACCOLADE trial of avacopan in C3 Glomerulopathy (C3G) and the AURORA trial of avacopan in Hidradenitis Suppurativa (HS). Our financial position is strong, enabling this historic period of data readouts. We look forward to a time soon where our innovations can be brought to the real service of our patients, to the aid of clinicians and to the benefit of those who have invested in this inventive enterprise."

Recent Highlights

Remained on track for mid-to-late Q4 topline data from the ADVOCATE global Phase III trial of avacopan in 331 patients with ANCA-associated vasculitis.

Completed enrollment in the Company’s LUMINA-1 Phase II randomized clinical trial of CCX140, an inhibitor of the chemokine receptor known as CCR2, in patients with sub-nephrotic primary Focal Segmental Glomerulosclerosis (FSGS), another rare kidney disease. Topline data is

anticipated in the first half of 2020. The single-arm, open label LUMINA-2 study continues to enroll, evaluating CCX140 in primary FSGS patients with the more severe nephrotic levels of proteinuria.

Awarded a two-year $1 million grant from the orphan drug office of the FDA to support advancement in the Company’s ACCOLADE Phase II clinical trial of avacopan in patients with the rare kidney disease C3 Glomerulopathy, a disease with no effective approved treatment. Approaching 60 percent enrollment in the ACCOLADE trial.

Attended Symposium on Hidradenitis Suppurativa Advances (SHSA), held November 1-3 in Detroit, Michigan. The Company’s AURORA Phase IIb clinical trial of avacopan for the treatment of the chronic disabling skin disease Hidradenitis Suppurativa (HS) is progressing well, with almost 80 percent of sites now activated and over 55 percent of patients enrolled to date.

Third Quarter 2019 Financial Results

Revenue was $10.6 million for the third quarter of 2019, compared to $9.0 million for the same period in 2018. Revenue is recognized based on the proportionate amount of costs incurred as a percentage of total budgeted costs to fulfill the performance obligations under the Company’s avacopan and CCX140 commercialization agreements with Vifor Pharma.

Research and development expenses were $18.1 million for the third quarter of 2019, compared to $15.1 million for the same period in 2018. The increase from 2018 to 2019 was primarily attributable to patient enrollment of the avacopan AURORA Phase II clinical trial in patients with HS and the two CCX140 LUMINA Phase II clinical trials in patients with FSGS, partially offset by decreases in research and drug discovery expenses.

General and administrative expenses were $6.1 million for the third quarter of 2019, compared to $5.4 million for the same period in 2018. The increase from 2018 to 2019 was primarily due to higher employee-related expenses, including those associated with our commercialization planning efforts, and higher professional fees.

Net loss for the third quarter of 2019 was $12.9 million, compared to $10.9 million for the same period in 2018.

Total shares outstanding at September 30, 2019 were approximately 58.3 million shares.

Cash, cash equivalents and investments totaled $205.8 million at September 30, 2019. The Company expects to close 2019 with cash and investments in excess of $185 million.

Conference Call and Webcast

The Company will host a conference call and webcast today, November 4, 2019 at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial (877) 303-8028 (Domestic) or (760) 536-5167 (International). The conference ID number is 5867903. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the conference call.