On May 29, 2020 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the publication of an electronic-poster (ePoster) with clinical proof-of-concept data from the Company’s phase 1b study in carfilzomib-refractory multiple myeloma patients treated with pelareorep in combination with carfilzomib (Kyprolis) (Press release, Oncolytics Biotech, MAY 29, 2020, View Source [SID1234558692]). Data presented in the ePoster demonstrates that the pelareorep-carfilzomib combination treatment results in selective replication of pelareorep in cancer cells and beneficial induction of an inflamed tumor environment associated with clinical responses. The ePoster was published this morning and will be presented this weekend as part of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Annual Meeting.

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"We are excited by the data showing an association between clinical and anti-tumor inflammatory response induced with pelareorep-carfilzomib treatment in this extremely difficult to treat patient population," said Dr. Douglas W. Sborov, MD, co-author. "The induction of cytokine release syndrome, which can be effectively monitored and managed via treatment with tocilizumab and steroids, is particularly interesting as it highlights the ability of the treatment to induce robust immune cell activation and tumor lysis. Taken together with earlier results from this study demonstrating pelareorep-induced upregulation of PD-L1 expression, the current data strongly support the potential of the ongoing trial investigating pelareorep, carfilzomib, and immune checkpoint inhibitor combination. This ongoing study could ultimately result in the development of a new treatment option for this high-need indication."

The ePoster, Oncolytic virus Pelareorep [P] plus Carfilzomib & Dexamethasone [Kd] phase 1 trial in Carfilzomib-refractory patients (NCI9603): responses with cytokine storm was co-authored by Dr. Douglas W. Sborov MD, MS, Assistant Professor, Division of Hematology and Hematologic Malignancies, University of Utah – Huntsman Cancer Institute, and Craig Hofmeister, M.D., MPH, Associate Professor, Department of Hematology and Medical Oncology Emory University School of Medicine, as well as several other colleagues at institutions across the United States. Key data and conclusions from six patients in the study are presented in the ePoster and include:

•Pelareorep targets and selectively replicates in multiple myeloma tumor cells
•Pelareorep, when combined with carfilzomib, activates a profound inflammatory response accompanied by a 50% ORR (overall response rate) and 83% CBR (clinical benefit rate)
•Three partial responses (PRs), one minimal response (MR), one stable disease (SD), and one progressive disease (PD) were achieved among patients with advanced and difficult-to-treat carfilzomib-refractory disease
•Significant and rapid T cell activation led to a single incidence of cytokine storm associated with tumor response after treatment with pelareorep and carfilzomib

"The exciting clinical proof-of-concept data demonstrate that pelareorep induces an inflammatory response in multiple myeloma, which is an unusual lymphoid tumor with immunosuppressive properties," said Dr. Rita Laeufle, Chief Medical Officer of Oncolytics Biotech. "The study examines the relationship between pelareorep-induced tumor inflammation and response to treatment. These findings reveal that induction of inflammation within the multiple myeloma tumor microenvironment may augment the effectiveness of checkpoint inhibitors, which to date have had little success against multiple myeloma."

The ePoster was presented as part of the Hematologic Malignancies-Plasma Cell Dyscrasia session at the ASCO (Free ASCO Whitepaper) Virtual Annual Meeting. A copy of the ePoster can be found on the Posters & Publications page of the company’s website: View Source

About Pelareorep
Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.

On May 29, 2020 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported the presentation of a poster highlighting the clinical trial design of its Phase 2 study of BP1001 (prexigebersen) at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place virtually from May 29-31, 2020 (Press release, Bio-Path Holdings, MAY 29, 2020, View Source [SID1234558691]).

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The poster, titled, "A Phase II Study of BP1001 (liposomal Grb2 antisense oligonucleotide) in Patients with Hematologic Malignancies," was presented virtually by Dr. Maro Ohanian, Department of Leukemia, University of Texas M.D. Anderson Cancer Center. The poster describes the Phase 2 study design of BP1001 (liposomal Grb2 antisense), the Company’s lead drug candidate, in combination with decitabine as a potential treatment for patients diagnosed with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS).

"This innovative trial design for BP1001 is unique in that it allowed us to adjust our treatment to include newly approved therapies that we believed would be enhanced from combination with our DNAbilize technology. We believe this robust design will provide for the best outcomes for patients and will be the most expeditious route to bringing BP1001 to market. We are delighted to have the design presented and expect that it will enhance visibility for our DNAbilize platform and its versatility among an audience dedicated to bringing new cancer treatments to patients," stated Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings.

The Phase 2 clinical trial is a multi-center, open label study with two parallel cohorts of BP1001 in combination with decitabine in untreated AML and high risk MDS patients or refractory/relapsed AML and high risk MDS patients who are ineligible or unwilling to receive intensive induction therapy. The primary objective of the study is to assess whether BP1001 in combination with decitabine provides higher response rates than decitabine alone in AML or high risk MDS patients. In addition, a six-patient safety run-in of BP1001 and decitabine was completed with no dose adjustment required.

BP1001 is a neutral liposome incorporated with nuclease-resistant, hydrophobic P-ethoxy antisense oligodeoxynucleotides targeted to Grb2 mRNA. Grb2 is an adaptor protein that links oncogenic tyrosine kinases with downstream kinases, such as ERK and AKT, which are critical to cell proliferation and survival. Preclinical results showed that BP1001 enhanced the inhibitory effects of cytarabine or decitabine against AML cells.

The poster also describes future development plans for BP1001 in AML. Preclinical results suggest that BP1001 plus venetoclax plus decitabine triple combination could be more efficacious than the BP1001 + decitabine combination against AML cells. Venetoclax will be added to the study, thus exploring three-drug combinations of BP1001, venetoclax and decitabine. There will be three patient cohorts in the study:

•Untreated AML patients will be treated with BP1001 plus venetoclax plus decitabine.
•Refractory/relapsed AML patients will be treated with BP1001 plus venetoclax plus decitabine.
•A third cohort of BP1001 + decitabine will be offered to refractory/relapsed AML patients who are venetoclax resistant or intolerant, or not considered by the investigator as optimal candidates for venetoclax-based therapy.

On May 29, 2020 Harpoon Therapeutics, Inc. (NASDAQ: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported interim data from the ongoing dose-escalation portion of a Phase 1 trial for HPN424 in patients with metastatic castration-resistant prostate cancer (mCRPC) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program (Press release, Harpoon Therapeutics, MAY 29, 2020, View Source [SID1234558690]). HPN424 targets prostate-specific membrane antigen (PSMA) and is based on Harpoon’s proprietary Tri-specific T cell Activating Construct (TriTAC) platform designed to recruit a patient’s own immune cells to kill tumor cells.

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The presentation highlights interim results in 44 patients across 11 dosing cohorts treated with HPN424 from the ongoing dose escalation portion of a Phase 1 clinical trial. As of the May 11, 2020 cut-off date, initial data demonstrate:

HPN424 is generally well-tolerated and support long-term treatment, and cytokine-related adverse events have been transient and manageable.

Pharmacokinetic data support weekly dosing and pharmacodynamic data supports T cell activation as measured by reduction in circulating tumor cells, increased serum cytokine levels and T cell margination after HPN424 administration.

Early signals of clinical activity include eight patients who remained on study treatment for greater than 24 weeks. In addition, eight patients exhibited decreases in PSA levels compared to baseline, including two who showed PSA (prostate-specific antigen) reductions of at least 50%.

"We are particularly encouraged by data supporting the predicted mechanism of action of HPN424, and the early signs of clinical activity in this heavily pretreated population," stated Gerald McMahon, Ph.D., President and CEO, Harpoon Therapeutics. "These initial data from our lead program represent the first of four product candidates in our TriTAC clinical portfolio that can provide multiple opportunities to accelerate our pipeline into more advanced clinical studies."

"We are excited to share the first clinical data from our HPN424 clinical program. Early data suggest that this novel, half-life extended T cell engager can be administered safely. Several patients remained on treatment for 24 weeks or more, which is notable in this late-stage cancer population," said Natalie Sacks, M.D., Chief Medical Officer of Harpoon. "We will continue dose escalation, and plan to open an expansion cohort later in 2020. Our goal is to develop an effective immunotherapy treatment option for patients with prostate cancer."

Trial Design and Interim Results from the HPN424 Phase 1 Clinical Trial

This Phase 1 trial is a multicenter, open-label study designed to evaluate the safety, tolerability, pharmacokinetics and activity of HPN424 in patients with mCRPC who are progressing at the time of enrollment and have had at least two prior systemic treatments for metastatic disease. The initial ongoing phase of the trial is dose escalation, with the goal of determining a recommended dose for the expansion phase. The escalation phase began with single patient cohorts and transitioned to a 3×3 design when Grade 2 toxicity was observed. HPN424 is being administered to patients once weekly by intravenous infusion. The primary outcome measures are an assessment of safety and tolerability, pharmacokinetics, and pharmacodynamics. Secondary endpoints include duration of response, progression free and overall survival. Tumor assessments include PSA, CT and bone scans performed every 9 weeks.

As of the May 11, 2020 cut-off date, 44 patients have been treated in 11 cohorts with doses ranging from 1.3 to 120 ng/kg. Enrolled patients had a median of 7 prior therapies, including 73% with prior chemotherapy, and a median of two prior novel hormonal agents. Median PSA level was 244, with a range of 0.1-5000 ng/ml. The most frequent adverse events were chills ((all grade n= 32 (73%), grade >3 n=0 (0%)), pyrexia (all grade n=21 (48%), grade > 3 n=0 (0%)), and cytokine release syndrome (CRS) (all grade n=14 (32%), grade >3 n=3 (7%)). Cytokine-related adverse events were transient, and all patients with these adverse events were retreated successfully with HPN424. Dexamethasone premedication was instituted in cohort 4 (24 ng/kg) and has been successfully administered to mitigate cytokine-related symptoms in patients treated with higher doses in subsequent cohorts. One DLT of asymptomatic Grade 3 serum lipase elevation was observed which resolved and the patient was retreated successfully as scheduled. The most common reasons for study discontinuation were due to progressive disease (72%) and unrelated adverse events (9%).

HPN424 demonstrated dose proportional increase in Cmax and AUC with a current estimate of median T1/2 of 24.9 hours (range: 9.0 – 312 hours). Dose-dependent, transient increases in peripheral cytokine and chemokine levels were observed, including increases in interleukin 6, peaking at 5 hours post infusion and returning to baseline 24 hours post-administration. Maximal cytokine/chemokine levels attenuated with each successive dose within six weeks. Baseline circulating tumor cells (CTC) ranged from 0-160 cells/ml of whole blood. Reduction in CTC was seen in 12 of 27 patients with evaluable CTC compared to baseline.

Eight of 26 patients (31%) with at least 24 weeks in follow-up remained on study beyond 24 weeks. Eight patients showed a PSA decline from baseline ranging from -4 to -76%, including patients with initial rises in PSA after study entry. Two patients had confirmed PSA partial responses with declines of 50% or greater.

Patients continue to be enrolled in the escalation phase of the trial, with a goal to identify a dose for an expansion phase planned for the second half of 2020. The expansion phase of the trial will further evaluate the safety and activity of HPN424 in patients with mCRPC. The trial is titled, "A Phase 1 Open-label, Multicenter, Dose Escalation and Dose Expansion Study of the Safety, Tolerability, and Pharmacokinetics of HPN424 in Patients with Advanced Prostate Cancer Refractory to Androgen Therapy". For additional information about the trial, please visit www.clinicaltrials.gov using the identifier NCT03577028.

Conference Call and Webcast Today

Harpoon’s management team will host a webcast and conference call today at 4 p.m. ET / 1 p.m. PT to review the ASCO (Free ASCO Whitepaper) data and provide an update on other pipeline programs. The live call may be accessed by dialing:

877-407-9716 for domestic callers

201-493-6779 for international callers

A live webcast of the call will be available from the Events and Presentations section of the company’s website at View Source and will be archived there shortly after the live event.

On May 29, 2020 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported updated data from the ongoing Phase 1 study evaluating single agent vorasidenib in isocitrate dehydrogenase (IDH)-mutant advanced solid tumors, including glioma (Press release, Agios Pharmaceuticals, MAY 29, 2020, View Source [SID1234558689]). Data from the non-enhancing glioma population were featured in an oral presentation at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, which is being held virtually. Vorasidenib, an investigational, oral, selective, brain-penetrant inhibitor of mutant IDH1 and IDH2 enzymes, is currently being evaluated in the registration-enabling Phase 3 INDIGO study as a potential treatment for patients with residual or recurrent Grade 2 non-enhancing glioma.

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"For patients with IDH-mutant non-enhancing glioma who currently have limited treatment options beyond chemotherapy and radiation, targeted oral options such as vorasidenib are urgently needed," said Ingo Mellinghoff, M.D., Memorial Sloan Kettering Cancer Center, an investigator for the Phase 1 dose-escalation study. "The updated results of this study in non-enhancing glioma patients provide further evidence of the potential benefit of vorasidenib for these patients, with a favorable safety profile and encouraging preliminary activity, including prolonged disease control, objective tumor responses, and clinically meaningful progression-free survival rates."

"These promising efficacy and safety data in patients with IDH-mutant non-enhancing glioma provide further support for our registration-enabling Phase 3 INDIGO study," said Chris Bowden, M.D., chief medical officer at Agios. "With vorasidenib – the first and only brain-penetrant IDH inhibitor in Phase 3 trials for low-grade glioma – we have an opportunity to target a highly prevalent driver mutation early in the disease evolution, providing a therapeutic alternative to ‘watch and wait’ that can potentially delay the need for chemotherapy and radiation."

Vorasidenib Phase 1 Dose-Escalation Study

Vorasidenib is being evaluated as a single agent in an ongoing Phase 1 dose-escalation trial in IDH1/2 mutant advanced solid tumors (n=93), including glioma (n=52). Enrollment was completed in June 2017. As of the March 3, 2020 data cut-off, study design, enrollment and baseline characteristics of the 22 non-enhancing glioma patients are reported below:

Seventy-seven percent of patients (n=17) had World Health Organization (WHO) classified Grade 2 tumors and 23% (n=5) had Grade 3 tumors.

Ninety-one percent of patients (n=20) had an IDH1 mutation and 5% (n=1) had an IDH2 mutation. One patient did not have a biopsy but was confirmed as IDH mutant positive due to 2-HG elevation by magnetic resonance spectroscopy (MRS).

The median age of these patients is 47 years (ranging from 16 to 73).

Sixty-four percent of patients (n=14) had received prior systemic therapy. Patients had received a median of two prior systemic therapies (ranging from 1 to 4).

Fifty-nine percent of patients (n=13) had previously received temozolomide and 36% (n=8) of patients received prior radiation therapy.

Patients received daily doses of vorasidenib ranging from 10 mg to 200 mg.

Thirty-six percent of patients (n=8) remain on treatment.

Safety Data

The safety analysis conducted on the 22 patients with non-enhancing glioma as of the data cut-off demonstrated that vorasidenib has a favorable safety profile at dose levels below 100 mg once daily. Safety data for this population are consistent with the results reported for all patients enrolled in this trial at the 2018 ASCO (Free ASCO Whitepaper) Annual Meeting.

The majority of adverse events (AEs) reported by investigators were mild to moderate, with the most common (>40%) across all grades being increased alanine aminotransferase (ALT) (64%), increased aspartate aminotransferase (AST) (59%), nausea (46%) and headache (41%).

Grade 3 or higher AEs were observed in 27% of patients (n=6) with the most common being increased ALT (9%) and AST (9%).

AEs of Grade 2 or higher elevated transaminases occurred in seven non-enhancing glioma patients at the higher dose levels (³100 mg) and resolved to Grade £1 with dose modification or discontinuation.

No AEs of Grade 2 or higher elevated transaminases were observed in patients at the lower dose levels (<100 mg).

Of the 14 (64%) patients who discontinued treatment, 9% (n=2) discontinued due to an AE.

Efficacy Data

Efficacy data from the 22 non-enhancing glioma patients as of the data cut-off showed:

The investigator-reported objective response rate (ORR) was 18% with one patient exhibiting a partial response and three patients exhibiting minor responses using the Response Assessment in Neuro-Oncology for low-grade glioma (RANO-LGG) criteria.

Seventy-three percent of patients (n=16) achieved stable disease according to the investigator as assessed by RANO-LGG.

With 59% of events reported, median progression free survival (PFS) was 31.4 months (95% CI 11.2, 40.8).

Twenty-four month PFS rate was 55.4%.

The median treatment duration was 25.8 months (ranging from 1.0 to 47.9) with 68% (n=15) remaining on treatment for ³1 year.

Ongoing Phase 3 INDIGO Trials in Progress Poster

A trials in progress poster was presented at the 2020 ASCO (Free ASCO Whitepaper) Annual Meeting to highlight the ongoing global, randomized, placebo-controlled Phase 3 INDIGO study of vorasidenib in approximately 366 patients with residual or recurrent, non-enhancing, Grade 2 low-grade glioma with an IDH1 or IDH2 mutation and who have undergone surgery as their only treatment. The goal of the study is to evaluate the efficacy of vorasidenib compared with placebo based on radiographic PFS and determine whether vorasidenib could provide a therapeutic alternative to "watch and wait" to help control low-grade glioma and potentially delay the need for chemotherapy and/or radiation. The study is currently enrolling. More information can be found on the INDIGO study website.

Vorasidenib is not approved in any country for the treatment of patients with low-grade glioma.

About Glioma

Glioma presents in varying degrees of tumor aggressiveness, ranging from slower growing (low-grade glioma) to rapidly progressing (high-grade glioma-Glioblastoma Multiforme). Tumor enhancement is an imaging characteristic assessed by magnetic resonance imaging (MRI), and enhancing tumors are more likely to be high-grade.

Common symptoms of glioma include seizures, memory disturbance, sensory impairment and neurologic deficits. The long-term prognosis is poor, and regardless of treatment, the majority of patients with low-grade gliomas will have recurrent disease that will progress over time. Approximately 11,000 low-grade glioma patients are diagnosed annually in the U.S. and EU and approximately 80 percent have an IDH mutation.

On May 29, 2020 MD Anderson reported that Patients with advanced non-small cell lung cancer (NSCLC) and the MET exon 14 (METex14) skipping mutation had a 46.5% objective response rate to the targeted therapy drug tepotinib, as shown in a study published today in the New England Journal of Medicine and presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Abstract 9556 – Poster 322) by researchers from The University of Texas MD Anderson Cancer Center (Press release, MD Anderson, MAY 29, 2020, View Source [SID1234558688]).

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"The success of this trial, alongside other studies on the same class of drugs, establishes MET exon 14 as an actionable target for non-small cell lung cancer," said senior author Xiuning Le, M.D., Ph.D., assistant professor of Thoracic-Head & Neck Medical Oncology. "We’re pleased to show that another group of lung cancer patients may benefit from precision medicine."

METex14 skipping is a mutation that drives cancer growth and occurs in 3-4% of all NSCLC patients. Patients with METex14 skipping tend to be older, with a median age of 74, and typically don’t have other actionable mutations with existing targeted therapy options.

The study results represent cohort A of the single-arm, international Phase II VISION trial, which is ongoing with additional cohorts. More than 6,700 NSCLC patients were prescreened for MET alterations through liquid and/or tissue biopsy. A total of 152 patients with advanced NSCLC and METex14 skipping were treated with tepotinib. Patients with prior treatment and/or stable brain metastasis were allowed to participate in the trial. Participants were treated with 500mg daily oral tepotinib.

Meaningful benefit for an elderly population

The primary endpoint was objective response rate, defined as complete or partial response, according to the RECIST v1.1 criteria and confirmed by independent review. After nine months follow-up, the primary efficacy population of 99 patients had a 46.5% objective response rate and durable response of 11.1 months.

"The median duration of response of almost one year is very meaningful for this patient population," Le said. "It’s important for these elderly patients to have another treatment option, other than traditional chemotherapy, in oral form that can improve their quality of life for a long duration."

Toxicities were manageable, with grade ≥ 3 treatment-related adverse events reported in 27.6% of patients. The most common side effect was peripheral edema. Eleven percent of patients discontinued treatment due to adverse events.

The study also collected patient-reported outcomes, which indicated an improvement in coughing and overall maintenance of quality of life.

Liquid biopsy for biomarker detection

The VISION study represents the largest METex14 skipping cohort to be identified prospectively through liquid biopsy, verifying that liquid biopsy is a reliable method to detect the mutation. The study also showed that liquid biopsy was a useful tool to identify response to the drug.

Matched liquid biopsy samples for baseline and on-treatment were available for 51 patients. Next-generation sequencing found 34 of those patients had a molecular response with a complete or deep reduction of the mutation, and radiographic response was confirmed in 68% of patients who had a molecular response.

"This study marked a major advance in that we now have a highly effective, oral therapy for a group of non-small lung cancer patients that previously did not have any targeted therapy options," said co-author John Heymach, M.D., Ph.D., chair of Thoracic-Head & Neck Medical Oncology. "We are proud to lead the field forward as we work to provide novel treatments to patients."

Tepotinib was granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) in September 2019, based on early data from the VISION study. It was approved for use as the first oral targeted therapy for MET-positive NSCLC in Japan in March 2020.

A full list of co-authors and their disclosures are included in the paper. The research was supported by Merck KGaA, Darmstadt, Germany.