Harpoon Therapeutics Announces Preliminary Safety and Pharmacology Data from its HPN424 Phase 1 Trial in Prostate Cancer

On January 2, 2019 Harpoon Therapeutics, Inc. ("Harpoon"), a clinical-stage immunotherapy company developing a novel class of T cell engagers that harness the power of the body’s immune system to treat patients with cancer and other diseases, reported preliminary safety and mechanism of action findings from an ongoing Phase 1 clinical trial evaluating its lead compound, HPN424, in patients with progressive metastatic castration resistant prostate cancer (mCRPC) (Press release, Harpoon Therapeutics, JAN 2, 2019, View Source [SID1234532362]). The Phase 1 trial is a multicenter, open-label dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics and clinical activity of HPN424, a TriTAC (Tri-specific T Cell Activating Construct) that targets prostate-specific membrane antigen (PSMA), a clinically validated tumor antigen target in prostate cancer.

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Eligible patients must have mCRPC, have received at least two prior treatment regimens for mCRPC, and have evidence of disease progression on the most recent systemic treatment regimen. Clinical observations from the first seven prostate cancer patients treated with HPN424 include:

Measurement of drug in the sera from patients indicates sufficient drug exposure during the treatment course to support once weekly dosing.
Measurable reduction in circulating tumor cells (CTC) in three of four patients evaluated as a biomarker for PSMA target engagement.
Dose-dependent, transient increase in peripheral cytokine and chemokine levels, consistent with the expected T cell activation mechanism of action.
No dose limiting toxicities have been observed to date, with four dose levels tested.
Adverse events were consistent with the expected mechanism of action, with three patients reporting grade 2 rigors or fevers that were manageable. A grade 3 cytokine release syndrome (CRS) event was reported in one patient from the fourth cohort but resolved within eight hours. All patients experiencing adverse events were successfully re-treated after one week without further complications.
All seven patients remain on study with weekly treatment of HPN424. Three patients have been analyzed for disease burden using computed tomography scans, bone scans and measurement of prostate serum antigen levels. To date, two patients have shown stable disease and one patient exhibited unconfirmed progressive disease and continues on study. The other four patients are early in their treatment and have not yet been evaluated for disease burden.
"These initial results in advanced metastatic castration resistant prostate cancer patients suggest that HPN424 is activating T cells in a manner that is consistent with tumor target engagement," said Natalie Sacks, M.D., Chief Medical Officer of Harpoon. Dr. Sacks added, "We have observed early signals confirming the mechanism of action of HPN424, and have obtained encouraging safety data that supports continued dose escalation to define a Phase 2 therapeutic dose and regimen."

"These results provide the first clinical evidence suggesting Harpoon’s TriTAC platform is performing as designed, by activating T cells in a target-dependent manner and by showing evidence of sufficient drug exposure during the treatment course to support once weekly dosing," said Holger Wesche, Ph.D., Chief Scientific Officer of Harpoon. "HPN424 is a novel T cell biologic that has been designed to deliver potent T cell killing to PSMA-expressing tumor cells. We are encouraged by the early human safety and pharmacology data emerging from our Phase 1 trial."

"We are excited by this early data for HPN424 and the broader application of our technology. Our novel TriTAC drug candidates are designed to enable a patient’s own T cells to fight tumors in a wide range of human malignancies," said Jerry McMahon, Ph.D., President and Chief Executive Officer of Harpoon. "We are looking forward to reporting additional clinical data from our ongoing HPN424 Phase 1 clinical trial in 2019 as we continue to enroll and analyze data from existing and new patients."

Phase 1 Trial Observations (As of December 31, 2018)

Patient Treatment and Tolerability: Seven patients have been treated in the dose escalation portion of the trial, at doses ranging from 1.3 to 24 ng/kg. All seven have been treated previously with a second-generation anti-androgen therapy. All seven patients remain on study treatment, with the time on treatment ranging from one to more than 20 weeks, and the number of doses received ranging from one to 22 weekly treatments. No dose limiting toxicities have been observed. One patient experienced a grade 3 CRS event which resolved within eight hours of dosing. This patient was re-administered HPN424 consistent with protocol guidelines. The patient experienced no further reactions and continues to be enrolled in the study. Based on this event, the dose escalation is proceeding using three to six patients per cohort consistent with the original Phase 1 plan.

Serum Drug Exposure: Preliminary pharmacokinetic analysis supports at least once weekly dosing. Maximum serum concentration of HPN424 was found to be proportional to the dose. The volume of distribution and clearance rates appear to be similar among different dose levels suggesting linear pharmacokinetic properties.

T Cell Engagement: Transient and dose-dependent increases in peripheral cytokines (interleukin-6, interleukin-8, interleukin-10) and chemokines (macrophage inflammatory protein-1-alpha, macrophage inflammatory protein-1-beta, monocyte chemoattractant protein-1) were observed, consistent with the expected mechanism of action related to T cell activation.

Tumor Assessments: Radiographic assessment of disease burden is performed at nine-week intervals. For the first three patients in the trial, radiographic and PSA data have shown stable disease for two patients and one patient exhibited unconfirmed progressive disease. The other four patients are early in their treatment and not yet evaluable. Whole blood samples from three of four patients with measurable baseline CTC levels showed a reduction in CTC following treatment with HPN424.

About HPN424-1001 Clinical Trial

The HPN424-1001 Phase 1 trial is a multicenter, open-label dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics and activity of HPN424, a novel T cell engaging therapeutic that targets PSMA (clinicaltrials.gov ID#: NCT03577028). Eligible patients must have mCRPC, have received at least two prior regimens for mCRPC, and have evidence of disease progression on the most recent systemic regimen. In the first part of the trial, HPN424 is administered once weekly via intravenous infusion in ascending dose cohorts until a therapeutic dose is achieved. Following dose escalation, the study will expand and enroll approximately 20 patients at the recommended Phase 2 dose established in the first part of the trial.

FLX Bio to Present at 37th Annual J.P. Morgan Healthcare Conference

On January 2, 2018 FLX Bio, Inc., a clinical-stage, biopharmaceutical company focused on the development of oral small-molecule drugs that target immune drivers of cancer and other immune-related disorders, reported that Brian Wong, M.D., Ph.D., CEO will present at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9, 2019 at 7:30 a.m. Pacific Standard Time in San Francisco, CA (Press release, FLX Bio, JAN 2, 2019, View Source [SID1234532361]). Dr. Wong will provide a corporate update and company overview.

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Deciphera Pharmaceuticals Announces Positive, Preliminary, Top-Line Clinical Data for the Ongoing Phase 1 Clinical Study with DCC-3014 and an Update on Future Development Plans

On January 2, 2018 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported positive, preliminary, top-line data from the ongoing dose escalation part of the Phase 1 clinical study with DCC-3014, the Company’s investigational small molecule switch control inhibitor of CSF1R, in patients with advanced malignancies (Press release, Deciphera Pharmaceuticals, JAN 2, 2019, View Source [SID1234532360]). In addition, the Company announced a plan to expand the Phase 1 study to evaluate DCC-3014 in patients diagnosed with Tenosynovial Giant Cell Tumors (TGCT). A review of further data from this Phase 1 study will be presented at a medical meeting in 2019.

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The Phase 1 study was designed to evaluate the safety, pharmacokinetics and pharmacodynamics of multiple doses of DCC-3014 in up to 55 patients.

As of the data cut-off date of November 9, 2018, increasing doses of DCC-3014 were assessed in five dose cohorts across 24 patients with advanced solid tumor malignancies. This included one dose cohort that received 10 mg once daily (QD) and four dose cohorts that followed a schedule of once or twice-weekly maintenance dosing preceded by a five-day loading dose regimen at doses of up to 30 mg per dose. In addition, four patients are currently enrolled in a dose cohort that will receive 40 mg twice weekly preceded by a five-day loading regimen.
Preliminary pharmacokinetic (PK) analysis showed dose-proportional exposure for DCC-3014 that we believe supports twice-weekly maintenance dosing preceded by a five-day loading dose regimen.
Biomarker data demonstrated strong target engagement of CSF1R, including material reductions in CSF1R positive macrophages in the blood that we believe constitutes mechanistic proof-of-concept (mPoC) for DCC-3014.
DCC-3014 was generally well tolerated in patients enrolled in the dose cohorts that received twice-weekly maintenance doses of DCC-3014 preceded by a five-day loading regimen at doses of up to 30 mg, which is summarized below:
Treatment emergent adverse events (TEAEs) were mostly Grade 1 or 2;
No Grade 3 or 4 DCC-3014 related TEAEs in ≥10% of patients;
No dose-limiting toxicities; and
A maximum tolerated dose has yet to be established.
In the dose-cohort that received 10 mg QD, clinically asymptomatic laboratory values were recorded as dose-limiting toxicities in two of seven patients.
"We are very pleased with the progress made to date in the Phase 1 clinical study with DCC-3014, our selective, and potent small molecule inhibitor of CSF1R," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "Based on the preliminary tolerability, PK, and mechanistic proof-of-concept data observed to date, we believe DCC-3014 is well suited for further development. While we will continue to enroll further patients in this Phase 1 study, we are planning to expand this study to enroll patients with tenosynovial giant cell tumors and to explore combinations with other therapies in the first half of 2019."

About DCC-3014

DCC-3014 is an investigational, orally administered, potent and highly selective inhibitor of CSF1R. DCC-3014 was designed using the Company’s proprietary switch control kinase inhibitor platform to selectively bind to the CSF1R switch pocket. DCC-3014 has greater than 100-fold selectivity for CSF1R over the closely related kinases FLT3, KIT, PDGFRα, PDGFRß and VEGFR2 and has an even greater selectivity for CSF1R over approximately 300 other human kinases. CSF1R controls the differentiation and function of macrophages including Tumor Associated Macrophages (TAMs) whose density within certain tumors including cancers of the breast, cervix, pancreas, bladder and brain correlates with poor prognosis. Tumors induce TAMs to suppress a natural immune response mediated by cytotoxic T-cells, a type of lymphocyte that would otherwise eradicate the tumor; a process known as macrophage checkpoints. Through inhibition of CSF1R DCC-3014 has in preclinical studies demonstrated potent macrophage checkpoint inhibition as both a single agent and in combination with PD1 inhibitors and other T-cell checkpoint inhibitors. DCC-3014 is currently being evaluated in a Phase 1 clinical study. For more information about the clinical trial design please visit www.clinicaltrials.gov (NCT03069469).

About Tenosynovial Giant Cell Tumors (TGCTs)

Tenosynovial giant cell tumors (TGCTs) are a group of benign tumors that involve the synovium, bursae and/or tendon sheath. Although benign, these tumors can grow and cause damage to surrounding tissues and structures inducing pain, swelling, and limitation of movement of the joint. Surgery is the main treatment option; however, these tumors tend to recur, particularly in pigmented villonodular synovitis, a diffuse-type of TGCT. If untreated or if the tumor continually recurs damage and degeneration may occur in the affected joint and surrounding tissues, which may cause significant disability. A genetic mutation in certain cells within the tumor causes overproduction of CSF-1, the ligand for the CSF1R receptor, which attracts macrophages and certain other cells that become the bulk of these tumors and cause the associated inflammatory changes.

AVEO Oncology to Present at the 37th Annual J.P. Morgan Healthcare Conference

On January 2, 2019 AVEO Oncology (NASDAQ:AVEO) reported that Michael Bailey, president and chief executive officer, will present at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 9, 2019 at 4:00 p.m. Pacific Time (Press release, AVEO, JAN 2, 2019, View Source [SID1234532359]).

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A live webcast of the presentation can be accessed by visiting the investors section of the Company’s website at www.aveooncology.com. A replay of the webcast will be archived for 30 days following the presentation date.

AVEO Oncology to Present TIVO-3 Data at the 2019 ASCO Genitourinary Cancers Symposium

On January 2, 2018 AVEO Oncology (NASDAQ: AVEO) reported that data from the Phase 3 TIVO-3 study of tivozanib (FOTIVDA) versus sorafenib in refractory advanced or metastatic renal cell carcinoma (RCC) will be presented during an oral session at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (GU) Cancers Symposium being held February 14-16, 2019 in San Francisco (Press release, AVEO, JAN 2, 2019, View Source [SID1234532358]).

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Presentation Details

Title: TIVO-3: A phase III, randomized, controlled, multicenter, open-label study to compare tivozanib to sorafenib in subjects with refractory advanced renal cell carcinoma (RCC)
Presenter: Brian Rini, MD, Cleveland Clinic Lerner College of Medicine of Case Western Reserve University
Abstract Number: 541
Session Title: General Session 8: Evolving Management of Metastatic Renal Cell Carcinoma
Data and Time: February 16, 2019, 10:00 a.m.-11:30 a.m. PT

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI) discovered by Kyowa Hakko Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union plus Norway and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3, and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC. Tivozanib has been investigated in several tumors types, including renal cell, hepatocellular, colorectal and breast cancers.