1stOncology™: Cancer Intelligence Service – Page 13193 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Epizyme to Present at the Wedbush PacGrow 2019 Healthcare Conference

On August 6, 2019 Epizyme, Inc. (Nasdaq:EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, reported that Robert Bazemore, president and chief executive officer, will present at the Wedbush PacGrow 2019 Healthcare Conference on Tuesday, Aug. 13, 2019 at 10:55 a.m. ET in New York City (Press release, Epizyme, AUG 6, 2019, View Source [SID1234538215]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

A live webcast will be available in the investor section of the company’s website at www.epizyme.com. The webcast will be archived for 60 days following the presentation.

Aeglea BioTherapeutics Reports Second Quarter 2019 Financial Results and Corporate Highlights

On August 6, 2019 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that engineers next-generation human enzymes to provide solutions for diseases with unmet medical need, reported financial results for the second quarter ended June 30, 2019 and corporate highlights (Press release, Aeglea BioTherapeutics, AUG 6, 2019, View Source [SID1234538214]).

"We made tremendous progress this quarter with pegzilarginase, our lead product candidate for the treatment of Arginase 1 Deficiency (ARG1-D)," said Anthony G. Quinn, M.B. Ch.B., Ph.D., president and chief executive officer of Aeglea. "We dosed our first patient in our global pivotal Phase 3 PEACE trial, which is a major milestone toward providing our therapy to patients. The U.S. Food and Drug Administration’s (FDA) breakthrough therapy designation highlights the clinical relevance of the emerging data from the Phase 1/2 and Phase 2 open-label extension clinical trials as well as the potential of pegzilarginase to provide meaningful clinical improvements over available therapy."

Recent Highlights

Aeglea dosed the first patient in the Company’s global pivotal Phase 3 PEACE trial. The pivotal trial is intended to further evaluate the efficacy and safety of pegzilarginase, the Company’s lead product candidate for the treatment of ARG1-D, a progressive disease presenting in early childhood that results in severe complications and early mortality. The Company expects to report topline data from the PEACE trial in the first quarter of 2021.

The FDA granted Breakthrough Therapy Designation (BTD) to the Company’s lead product candidate, pegzilarginase, for the treatment of ARG1-D. The FDA’s BTD is intended to expedite the development and review of new therapies that are aimed at treating a serious or life-threatening condition when preliminary clinical evidence demonstrates the therapy may have substantial improvement on at least one clinically significant endpoint over available therapy. The designation was based on data from the completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study. Aeglea expects to continue discussions with the FDA regarding the pegzilarginase program and the Company’s next steps in the fourth quarter of 2019.

Interim data from 35 patients in the Company’s Phase 1/2 combination trial of pegzilarginase and KEYTRUDA in extensive disease small cell lung cancer revealed to date one complete response, four partial responses and 11 patients with stable disease. The combination trial was well tolerated, and safety observations were consistent with prior studies of pegzilarginase in patients with cancer. The Company has concluded enrollment and intends on submitting the results for presentation or publication after the final dataset becomes available.
Upcoming Events

Aeglea will present at the following conferences, with details regarding the date and time of the presentations and webcasts to be announced prior to the events.

Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, September 3-6, Rotterdam, Netherlands
H.C. Wainwright & Co. 21st Annual Global Investment Conference, September 8-10, New York, NY
Second Quarter 2019 Financial Results

As of June 30, 2019, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $107.0 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.

Research and development expenses totaled $14.8 million for the second quarter of 2019, compared with $9.1 million for the second quarter of 2018. The increase was primarily due to expanded personnel-related expenses, clinical development activity, investment in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase, and a ramp-up in manufacturing activities for the Company’s AEB4104 program for homocystinuria.

General and administrative expenses totaled $3.8 million for the second quarter of 2019, compared with $2.9 million for the second quarter of 2018. This increase was primarily due to additional employee headcount and compensation to support company growth.

Net loss totaled $18.0 million and $9.4 million for the second quarter of 2019 and 2018, respectively, with non-cash stock compensation expense of $1.2 million and $1.0 million for the second quarter of 2019 and 2018, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically depletes the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. Aeglea’s Phase 1/2 and Phase 2 open-label extension data evaluating pegzilarginase in patients with Arginase 1 Deficiency demonstrated clinical improvements and sustained lowering of plasma arginine. Aeglea is currently recruiting patients for its single, global pivotal Phase 3 PEACE trial designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

Blueprint Medicines to Present at 2019 Wedbush PacGrow Healthcare Conference

On August 6, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported that Andy Boral, M.D., Ph.D., Blueprint Medicines’ Chief Medical Officer, will present a company overview at the 2019 Wedbush PacGrow Healthcare Conference in New York, NY on Tuesday, August 13, 2019 at 9:45 a.m. ET (Press release, Blueprint Medicines, AUG 6, 2019, View Source [SID1234538213]).

A live webcast of the presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.

Oncternal Therapeutics Announces Opening of Randomized Phase 2 Study of Cirmtuzumab in Combination with Ibrutinib

On August 6, 2019 Oncternal Therapeutics, Inc., (Nasdaq: ONCT) a clinical-stage biotechnology company developing potential first-in-class product candidates for cancers with critical unmet medical need, reported that it has opened for enrollment its randomized Phase 2 study of cirmtuzumab, a ROR1-targeted monoclonal antibody, combined with ibrutinib in patients with chronic lymphocytic leukemia (CLL) (Press release, Oncternal Therapeutics, AUG 6, 2019, View Source [SID1234538212]). The decision to open Phase 2 of the company’s ongoing Phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial was triggered by favorable outcomes from the Part 1 dose-finding and Part 2 dose-confirming cohorts of the clinical trial, including an observed interim objective response rate (ORR) of 100% for the first 9 CLL patients with evaluable data receiving the recommended dosing regimen who have completed 12 weeks of cirmtuzumab plus ibrutinib treatment in Part 2, and a well-tolerated safety profile consistent with that seen with ibrutinib treatment alone.

In June, the company presented data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, reporting that results from the first 12 patients with CLL treated in Part 1 of the Phase 1 portion of the study showed an observed interim objective response rate (ORR) of 91.7% for the combination of cirmtuzumab plus ibrutinib, including three patients with clinical or confirmed complete responses, and a well-tolerated safety profile consistent with that seen for ibrutinib treatment alone.

Included in the results presented at ASCO (Free ASCO Whitepaper) were preliminary results from six patients with mantle cell lymphoma (MCL), who were treated in a separate cohort of the CIRLL study. Data from this cohort will be presented at a future medical conference. One patient with MCL who had relapsed following an allogeneic stem cell transplant experienced a confirmed complete response (CR) after 3 months of cirmtuzumab plus ibrutinib treatment, including complete resolution of a large mediastinal mass. This CR appears to be durable, and has been confirmed after 6, 9 and 11 months of cirmtuzumab plus ibrutinib treatment.

The CIRLL clinical trial is supported by a grant from the California Institute for Regenerative Medicine (CIRM) and is being conducted in collaboration with the University of California at San Diego (UC San Diego).

"We are very pleased to be opening the randomized Phase 2 portion of the CIRLL study for patients with CLL and continue to be encouraged by the interim results from the study for both patients with CLL and patients with mantle cell lymphoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO.

About the Study
The CIRLL Study (CIRM-0001) is a Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib in patients with CLL or MCL. Part 1 of the study was a Phase 1 dose-finding portion designed to determine the Phase 2 dose, or recommended dosing regimen (RDR). Part 2 was a Phase 1b expansion cohort to confirm the RDR. Interim analyses were specified for Part 1 and Part 2. Part 3 of the study, which is now open for enrollment, is a Phase 2 study in which approximately 90 patients with CLL will be randomized to receive either ibrutinib alone or ibrutinib plus cirmtuzumab, with a primary endpoint of complete response rate. An interim assessment of the first 12 patients with CLL enrolled in Part 1 of the study was presented as a poster at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting. Additional information about the CIRM-0001 study and other clinical studies of cirmtuzumab may be accessed at ClinicalTrials.gov.

About Cirmtuzumab
Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of CLL and MCL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine. In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with metastatic breast cancer is being conducted at the UC San Diego School of Medicine. CIRM has also provided funding to support development programs for cirmtuzumab and a CAR-T product candidate that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors.

When expressed by hematologic malignancies such as CLL and MCL, ROR1 acts as a receptor for the tumor growth factor Wnt5a. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to inhibiting Wnt5a activation, specifically targeting ROR1 expressing tumors, and this finding led to the discovery of the potent and highly selective antitumor activity of cirmtuzumab observed in preclinical studies. Oncternal believes ROR1 is an attractive target for cancer therapy because it is an oncofetal antigen – a protein not normally expressed in adults, but which confers a survival and fitness advantage when reactivated and expressed by tumor cells. Preclinical data indicate that when cirmtuzumab binds to ROR1, it blocks Wnt5a signaling, induces differentiation of the tumor cells, and inhibits tumor cell proliferation, migration and survival. Cirmtuzumab is in clinical development and has not been approved by the U.S. Food and Drug Administration for any indication.

Can-Fite to Treat Advanced Liver Cancer Patients with Namodenoson Under Compassionate Use Setting in Israel

On August 6, 2019 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported that a supply of Namodenoson has been manufactured and is ready for use in the treatment of advanced liver cancer patients under compassionate use at the Rabin Medical Center in Israel (Press release, Can-Fite BioPharma, AUG 6, 2019, View Source [SID1234538211]).

Compassionate use allows doctors and their patients the option of early access to investigational new drugs, under closely controlled and monitored circumstances, when a patient who is facing serious illness has exhausted all available treatment options.

Salomon M. Stemmer, MD, the Principal Investigator of the Company’s prior Phase II liver cancer study said, "Given the evidence of clinical benefit of Namodenoson in patients with hepatocellular carcinoma and Child Pugh B7 to whom there is no accepted well established treatment, I plan to offer Namodenoson to certain HCC CPB7 patients in the compassionate use setting."

Can-Fite recently announced results from its Phase II study of Namodenoson in the treatment of advanced liver cancer. Namodenoson was found to increase overall survival in HCC patients with Child Pugh B7, the largest subpopulation of the study, as compared to placebo, even though the trial did not meet its primary endpoint.

An end of Phase II meeting with the U.S. Food and Drug Administration to review study data and to present the design of a Phase III clinical trial is expected soon. The FDA has granted Namodenoson both Orphan Drug and Fast Track status providing a pathway for accelerated approval based on unmet need in the treatment of advanced liver cancer. Fast Track designation offers advantages including more frequent meetings with the FDA and rolling review, which provides the opportunity to submit parts of its New Drug Application (NDA) for review prior to completing the entire application for commercialization. Orphan Drug designation includes 7-year market exclusivity following marketing approval, FDA assistance during the drug development process, and exemption of application fees.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.