Rainier Therapeutics Announces Results from First Interim Analysis of the FIERCE-22 Trial of Vofatamab in Combination with Pembrolizumab in Patients with Metastatic Urothelial Cell Carcinoma (Bladder Cancer)

On June 3, 2019 Rainier Therapeutics, Inc., a privately held clinical stage drug development company, reported the first interim analysis results from its ongoing FIERCE-22 trial of vofatamab in patients with metastatic bladder cancer (Press release, Rainier Therapeutics, JUN 3, 2019, View Source [SID1234536840]). Results were presented at the 2019 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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FIERCE-22 is a Phase 1b/2 trial evaluating vofatamab, a FGFR3-targeted antibody, in combination with pembrolizumab, an immune checkpoint inhibitor, to determine safety, tolerability and preliminary efficacy in the treatment of patients with locally advanced or metastatic bladder cancer who have progressed following platinum-based chemotherapy and who have not received prior immune checkpoint inhibitor therapy.

The first interim analysis included data from 28 patients enrolled in the Phase 2 trial, which has a targeted total enrollment of approximately 74 patients. Results demonstrate that treatment with vofatamab and pembrolizumab was well tolerated. Five patients discontinued the study due to treatment emergent adverse events, none related to vofatamab. No dose reductions of vofatamab occurred.

An overall best response rate of 36 percent (8/22) was observed in patients with lesions evaluable by RECIST1.1 criteria, 7/22 (32%) were confirmed. Response rates were similar in both wild type and mutant fusion patients (33% and 43%, respectively). A majority of patients have received at least 8 cycles of therapy (1 cycle=21 days). Responses were notably increased in patients with luminal biology (6 of 9 patients). Paired biopsy data from this trial were recently presented at AACR (Free AACR Whitepaper) "Frontiers in Bladder Cancer" (see poster) showing vofatamab induced immune and inflammatory changes in patients with responses.

"These data provide very encouraging clinical evidence of the effect of vofatamab, an antibody targeted against both wild type and mutated FGFR3, in combination with pembrolizumab. Earlier results from this biopsy-driven trial suggested these responses in the wild type FGFR3 patient cohort occurred in urothelial cancers of luminal subtype which typically have immunologically cold tumors. Biopsies obtained post lead-in treatment with vofatamab showed upregulation of genes associated with an inflammatory response," said Arlene O. Siefker-Radtke, MD, The University of Texas MD Anderson Cancer Center. "Further studies are needed to understand the impact of FGFR3 inhibition in urothelial cancer."

"The evidence demonstrated vofatamab provided similar clinical benefits in these patients regardless of FGFR3 tumor status. We plan to continue the enrollment of both wild-type and mutant/fusion patients, and to complete the ongoing Phase 2 trial," said Esteban Abella, MD, Chief Medical Officer of Rainier Therapeutics.

In addition, results were presented from the Phase 2 portion of the FIERCE-21 trial evaluating vofatamab in combination with docetaxel and vofatamab as monotherapy in patients with locally advanced or metastatic bladder cancer with FGFR3 mutations or gent fusions who have relapsed after or are refractory to at least one prior line of chemotherapy. Data presented indicated vofatamab alone and in combination was well tolerated, with no observed long-term safety issues. Vofatamab monotherapy demonstrated single-agent activity in heavily pre-treated patients. In addition, a substantial portion of patients demonstrated long-term benefit from monotherapy and combination therapy.

About Vofatamab

Vofatamab (formerly B-701) is an antibody specifically targeted against the fibroblast growth factor receptor 3 (FGFR3), a known driver of bladder and potentially other FGFR-driven cancers. Vofatamab is the most advanced targeted antibody specific for FGFR3 known by Rainier Therapeutics to be in clinical development. Vofatamab is currently being evaluated in two clinical trials: FIERCE-22 and FIERCE-21.

Aadi Bioscience Breakthrough Therapy nab-Sirolimus (ABI-009) Preliminary Data Released from the AMPECT Registration Trial in Advanced PEComa

On June 3, 2019 Aadi Bioscience, Inc (Aadi), a privately held clinical stage biopharmaceutical company,reported preliminary data on the ongoing nab-sirolimus (ABI-009) registration trial (AMPECT) for Advanced (metastatic or locally advanced) Malignant PEComa (perivascular epithelioid cell tumor) – a rare form of sarcoma for which there is no currently approved therapy (Press release, Aadi, JUN 3, 2019, View Source [SID1234536839]).

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Nanoparticle albumin-bound sirolimus (nab-Sirolimus), an mTOR inhibitor, received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA) in Dec 2018, "for the treatment of patients with advanced (metastatic or locally advanced) malignant perivascular epithelioid cell tumor (PEComa)."

The AMPECT trial was conducted at 9 U.S. sites and enrolled 34 adult patients, with 31 confirmed as PEComa by a central pathology laboratory. PEComa origin sites in these patients included the uterus, pelvis, retroperitoneum, lung, kidney, liver, brain, muscle, ovary, aorta and small bowel.

These data, demonstrating a 42 percent confirmed investigator-assessed objective response rate (ORR) across advanced PEComas originating in various tissues, were released in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 annual meeting in Chicago (abstract 11005). AMPECT Principal Investigator Andrew Wagner, M.D., Ph.D., from the Dana Farber Cancer Institute, said nab-sirolimus had delivered consistent and durable responses in advanced PEComa patients. "These responses were achieved with a manageable safety profile," Dr. Wagner said. "We saw a majority of the responding patients achieving a response by their first assessment at 6 weeks following initiation of therapy. Cytotoxic chemotherapies and other drugs approved for treatment of advanced sarcomas show only marginal benefit in PEComas. Activation of the mTOR pathway is common in PEComa and case reports have shown activity of mTOR inhibitors [1]. We are encouraged by the outcomes in this first-ever prospective clinical trial in advanced PEComa."

"The Aadi Bioscience team is proud to have contributed to this important study presented at ASCO (Free ASCO Whitepaper)," said Neil Desai, Ph.D., Chief Executive Officer of Aadi Bioscience. "We are grateful to the patients, families, and clinical trial teams who help push the boundaries of available care through their participation in clinical trials. These results are an important milestone in the ongoing development of nab-sirolimus across a wide range of diseases and therapeutic indications that are driven by mTOR activation and for which there is a need for new therapies." Dr. Desai added: "Results from the AMPECT study will serve as the basis for the ABI-009 New Drug Application (NDA) for nab-sirolimus, which the company expects to submit to the FDA in late 2019 or early 2020. The primary analysis for the NDA will rely upon central, independent radiology review, which will be performed in the second half of 2019. The company plans to release these data at a scientific meeting, which will also include additional patient follow-up, before the end of 2019."

Key Data Presented at ASCO (Free ASCO Whitepaper)

The primary efficacy outcome measure for the analysis presented at ASCO (Free ASCO Whitepaper) is investigator-assessed objective response rate (ORR) as measured by RECIST v 1.1. Key secondary endpoints include duration of response (DOR), progression-free survival (PFS), PFS rate at 6 months (PFS6) and safety. The data presented at ASCO (Free ASCO Whitepaper) employed a May 10, 2019 data cut-off, summarized below, was based on response assessments as performed by each respective clinical trial site (local, investigator-assessed radiology). A separate response assessment performed by independent radiologists, not yet completed, will be required to support global regulatory filings.

Consistent with agreements with the FDA, advanced PEComa patients enrolled in the Phase 2 trial (AMPECT) contributed to the efficacy analysis. The data presented are based on 34 patients evaluable for safety and 31 patients evaluable for efficacy per defined criteria in the protocol.


Enrolled Patients with Confirmatory Response Data Available (n = 31)
Objective Response Rate
(ORR = all PRs) 42% (95% CI: 25% – 61%)
Stable Disease 35%
Disease Control Rate (PR+SD) 77%
Progressive Disease 23%

Sixty-two percent of patients (8/13) with responses are continuing on treatment which include 3 patients on therapy for more than 1 year and 3 patients on therapy for more than 2 years. Median DOR has not been reached; median time to response is 1.4 months (95% CI: 1.3, 2.7). Median PFS is 8.4 months (95% CI: 5.5, –), PFS rate at 3 months (PFS3) is 80% (95% CI: 60%, 90%), PFS6 is 61% (95% CI: 41%, 76%), and 32 percent of all patients enrolled remain on treatment.

For reference, per a meta-analysis of 10 years of phase 2 trials in advanced soft tissue sarcomas (STS) published by the EORTC STS and Bone Sarcoma Group [2], the PFS3 and PFS6 are widely accepted as a meaningful measure of activity of drugs in STS and may be utilized to determine acceptable criteria of benefit. Drugs yielding a PFS rate of ≥40% at 3 months and ≥14% at 6 months are considered to be ‘potentially active’ in advanced STS [2].

A protocol prespecified exploratory mutational and biomarker analysis was available for 25 patients on the AMPECT trial. Mutational status of the suspect genes TSC1 or TSC2 in the mTOR pathway were analyzed for association with patient response outcomes. Mutation or deletion of TSC1 or TSC2 (no overlap) occurred in 5 (20%) and 9 (36%) patients respectively, while 11 (44%) patients had no alterations in TSC1 or TSC2. Responses occurred in 9/9 (100%) patients with TSC2 mutations, 1/5 (20%) patients with TSC1 mutations and 1/11 (9%) of patients with no mutations in TSC1 or TSC2.

The safety data presented at ASCO (Free ASCO Whitepaper) was available for all 34 patients treated on the AMPECT trial. The most common treatment-related hematologic adverse events of any grades included anemia (47%) and thrombocytopenia (32%) and the most common nonhematologic treatment-related adverse events of any grades included mucositis (74%), rash (65%), fatigue (59%), nausea (47%), and diarrhea (38%). Most of these events were grade 1 and 2, were manageable with dose modifications and no grade 4 events were observed. Twelve patients (35%) required dose reductions due to adverse events. Two patients (6%) discontinued nab-sirolimus due to an adverse event.

The AMPECT Phase 2 registration trial for Advanced Malignant Perivascular Epithelioid Cell Tumors completed enrollment in late 2018. Aadi previously received agreement from the FDA that this open label study in approximately 30 efficacy evaluable patients with a primary endpoint of independently reviewed objective response rate could support the submission of an NDA for approval to treat this rare disease.

Imbruvica®▼ (ibrutinib) Long-Term Data from Two Pivotal Phase 3 Studies at ASCO and EHA Demonstrate Sustained Efficacy and Safety in Patients with Chronic Lymphocytic Leukaemia (CLL)

On June 3, 2019 The Janssen Pharmaceutical Companies of Johnson & Johnson reported long-term follow-up results from two pivotal Phase 3 studies of Imbruvica (ibrutinib) in patients with chronic lymphocytic leukaemia (CLL), a type of non-Hodgkin lymphoma and the most common form of leukaemia in adults.3 One set of data – results from the RESONATETM study (PCYC-1112) at a median follow-up of 65.3 months (range, 0.3–71.6) – showed treatment with ibrutinib monotherapy sustained progression-free survival (PFS) benefit compared to ofatumumab in patients with previously treated CLL, with a median PFS of 44.1 months versus 8.1 months, respectively.1 A consistent PFS benefit with ibrutinib was observed across all baseline disease and patient characteristics, including patients with genomically defined high-risk disease.1 The median overall survival (OS) was 67.7 months in the ibrutinib arm and 65.1 months in the ofatumumab arm, without censoring or adjustment for crossover from ofatumumab to ibrutinib.1 Additionally, no new safety events were identified in this long-term follow-up.1 The RESONATETM results were presented today at the 55th American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, and selected for the Best of ASCO (Free ASCO Whitepaper) 2019 Meetings, which highlight cutting-edge science and reflect leading research in oncology (abstract #7510).

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The second data set – results from the RESONATETM-2 study (PCYC-1115/1116) at a median follow-up of five years (range, 0.1–66 months) – demonstrated durable PFS with ibrutinib monotherapy (estimate of 70 percent) versus chlorambucil (estimate of 12 percent) in patients with previously untreated CLL, including those with genomically defined high-risk disease.2 The OS benefit was also sustained in patients treated with ibrutinib (estimate of 83 percent) versus chlorambucil (estimate of 68 percent). In addition, no new safety concerns were observed.2 The RESONATETM-2 data will be presented in full during an oral presentation at the 24th European Hematology Association (EHA) (Free EHA Whitepaper) Congress in Amsterdam on Friday, June 14 (abstract #S107).2

"Since its first European approval in 2014, ibrutinib has redefined treatment paradigms for CLL, and these study results offer further evidence to both clinicians and patients of the longer-term benefits and tolerability ibrutinib offers as a single agent," said Peter Hillmen, MB ChB, PhD, Professor of Experimental Haematology and Honorary Consultant Haematologist at Leeds Teaching Hospitals NHS Trust, United Kingdom and investigator in both studies. "Not only is superior progression-free survival and overall survival maintained with ibrutinib follow-up, but frequently the quality of response rates improves from partial to complete over time."

"Ibrutinib has already impacted more than 140,000 patients, and the RESONATE and RESONATE-2 long-term follow-up studies provide important data in support of its continued use in the effective management of CLL," said Dr Patrick Laroche, Europe, Middle East and Africa (EMEA) Haematology Therapeutic Area Lead, Janssen-Cilag France. "We are excited to explore how best this BTK inhibitor can continue to enhance the lives of people living with CLL, both as a monotherapy and in newer combination regimens, and as an alternative option to intensive chemotherapy."

Ibrutinib, a Bruton’s tyrosine kinase (BTK) inhibitor, is jointly developed and commercialised by Janssen Biotech, Inc., and Pharmacyclics LLC, an AbbVie company.

ASCO: RESONATETM six-year follow-up of ibrutinib monotherapy in patients with previously treated CLL (Abstract #7510)1

The RESONATETM (PCYC-1112) study evaluated patients with previously treated CLL who were randomised to receive ibrutinib 420 mg orally once daily until disease progression or intravenous ofatumumab for up to 24 weeks (n=391); 86 percent and 79 percent, respectively, were in the genomically defined high-risk population (17p deletion, 11q deletion, TP53 mutation, and/or unmutated IGHV).1 Long-term efficacy endpoints were investigator-assessed.1

With up to six years of follow-up (median 65.3 months, range, 0.3–71.6 months), extended ibrutinib treatment showed sustained efficacy in patients with previously treated CLL, including patients with high-risk genomic features, with no new safety signals over long-term therapy.1

Of the patients receiving ofatumumab, 68 percent crossed over to receive ibrutinib.1 A statistically significant PFS benefit was sustained with ibrutinib versus ofatumumab, with median PFS of 44.1 months versus 8.1 months (hazard ratio [HR]=0.15; 95 percent confidence interval [CI], 0.11–0.20, P˂0.0001), and was consistent across baseline subgroups.1 Median PFS in the genomically defined high-risk population was 44.1 months versus 8.0 months on ibrutinib versus ofatumumab (HR=0.11; 95 percent CI, 0.08–0.15).1

The median OS was 67.7 months in the ibrutinib arm and 65.1 months in the ofatumumab arm, without censoring or adjustment for crossover from ofatumumab to ibrutinib (HR=0.81; 95 percent CI, 0.60-1.09).1 Sensitivity analysis adjusting for crossover based on the rank-preserving structural failure time (RPSFT) method also showed continued OS benefit with ibrutinib compared to ofatumumab (HR=0.24; 95 percent CI, 0.11-0.55).1 The overall response rate (ORR) with ibrutinib was 91 percent, with 11 percent achieving a complete response (CR/CR with incomplete blood recovery [CRi]).1 Median treatment duration of ibrutinib was 41 months; 40 percent of patients received ibrutinib for longer than four years.1

The adverse event (AE) profile with ibrutinib remained consistent with prior studies.1 The prevalance of any Grade 3 or higher AEs with ibrutinib decreased after the first year and remained stable thereafter. All Grade and Grade 3 or higher AEs, respectively, included hypertension (21 percent; 9 percent) and atrial fibrillation (12 percent; 6 percent); major haemorrhage occurred in 10 percent.1,4 The most common reasons for ibrutinib discontinuation prior to study closure were progressive disease (37 percent) and AEs (16 percent).1

EHA: RESONATETM-2 five-year follow-up of ibrutinib monotherapy in patients with previously untreated CLL (Abstract #S107)2

The RESONATETM-2 (PCYC-1115/1116) study evaluated patients 65 years or over with previously untreated CLL, without 17p deletion, who received ibrutinib 420 mg orally once-daily continuously until disease progression or unacceptable toxicity, or chlorambucil 0.5–0.8 mg/kg orally for up to 12 cycles (n=269).2

Results from this five-year follow-up showed ibrutinib monotherapy sustained PFS and OS benefits for patients with CLL versus chlorambucil, including those with high-risk genomic features.2 More than half of patients remain on long-term continuous treatment with ibrutinib. Additionally, no new safety concerns were identified.2

At a median follow-up of 60 months (range, 0.1–66 months), the PFS benefits were sustained in patients treated with ibrutinib (estimate of 70 percent) versus chlorambucil (estimate of 12 percent) (HR=0.15; 95 percent CI, 0.10–0.22).2 The OS benefits were also sustained in patients treated with ibrutinib (estimate of 83 percent) versus chlorambucil (estimate of 68 percent).2 Ibrutinib improved PFS compared with chlorambucil in patients with unmutated IGHV (HR=0.11; 95 percent CI, 0.06–0.19) and in patients with 11q deletion (HR=0.03; 95 percent CI, 0.01–0.11).2 Additionally, 57 percent of patients crossed over from chlorambucil to ibrutinib after progression.2

As a composite, patients with high-risk genomics (unmutated IGHV, 11q deletion, and/or TP53 mutation) had superior outcomes with ibrutinib compared with chlorambucil (PFS: HR=0.08; 95 percent CI, 0.05–0.15; OS: HR=0.37; 95 percent CI, 0.18–0.74).2 With ibrutinib, the ORR including partial response with lymphocytosis was 92 percent and the CR/CRi rate increased over time to 30 percent (from 11 percent CR/CRi at primary analysis at median follow-up of 18 months).2

The most common Grade 3 or higher AEs included neutropenia (13 percent), pneumonia (12 percent), hypertension (8 percent), anaemia (7 percent), hyponatremia (6 percent), atrial fibrillation (5 percent), and cataract (5 percent), with rates of most events decreasing over time.2 Dose reductions due to Grade 3 or higher AEs decreased over time. Benefit with ibrutinib treatment continued in 58 percent of patients who remained on therapy at the time of this analysis.2

#ENDS#

About ibrutinib

Ibrutinib is a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor, which works by forming a strong covalent bond with BTK to block the transmission of cell survival signals within the malignant B-cells.5 By blocking this BTK protein, ibrutinib decreases survival and migration of B lymphocytes, thereby delaying the progression of the cancer.6

Ibrutinib is currently approved in Europe for:7

Chronic lymphocytic leukaemia (CLL): As a single agent for the treatment of adult patients with previously untreated CLL, and as a single agent or in combination with bendamustine and rituximab (BR) for the treatment of adult patients with CLL who have received at least one prior therapy.
Mantle cell lymphoma (MCL): Adult patients with relapsed or refractory mantle cell lymphoma.
Waldenström’s macroglobulinemia (WM): Adult patients who have received at least one prior therapy or in first-line treatment for patients unsuitable for chemo-immunotherapy.
Ibrutinib is approved in more than 90 countries, and, to date, has been used to treat more than 140,000 patients worldwide across its approved indications.

The most common adverse reactions seen with ibrutinib include diarrhoea, neutropenia, haemorrhage (e.g., bruising), musculoskeletal pain, nausea, rash, and pyrexia.7

For a full list of side effects and information on dosage and administration, contraindications and other precautions when using ibrutinib please refer to the Summary of Product Characteristics for further information.

CEL-SCI Corporation to Present at the 9th Annual LD Micro Invitational

On June 3, 2019 CEL-SCI Corporation (NYSE American: CVM), a Phase 3 cancer immunotherapy company, reported that it will be presenting at the 9th annual LD Micro Invitational on Tuesday, June 4th at 10:40 a.m. PT. Geert Kersten, Chief Executive Officer of CEL-SCI, will be giving the presentation (Press release, Cel-Sci, JUN 3, 2019, View Source [SID1234536837]).

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Mr. Kersten’s presentation will be webcast and available in the Investor Relations section of the Company’s website at View Source The webcast will be archived for 90 days following the presentation.

The conference will be held at the Luxe Sunset Boulevard Hotel in Los Angeles, California, will feature 250 companies in the small-cap / micro-cap space, and will be attended by over 1,000 individuals.

GeneCentric Therapeutics Presents First Data on Novel Response Signatures to Treatment in Muscle Invasive Bladder Cancer (MIBC) Patients

On June 3, 2019 GeneCentric Therapeutics reported that it will present the first data on novel response signatures for metastatic, muscle invasive bladder cancer (MIBC) treatments by applying its proprietary, RNA-sequencing based predictive response signature and Cancer Subtyping Platform (CSP) (Press release, GeneCentric Therapeutics, JUN 3, 2019, View Source [SID1234536836]). The initial response signature and MIBC cohort data are being presented at a poster session at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago, IL, later today.

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"MIBC remains a significant area of unmet need for patients, with a median 5-year survival rate of 5 percent," said Dr. Mike Milburn, CEO/President of GeneCentric and senior author on the study. "The advent of immune checkpoint inhibitors (ICP) offers further treatment options in MIBC, however, their clinical benefit varies considerably by patient. We are excited to be at the forefront of advancing such gene response signatures for the benefit of patients."

The overall goal of the study, conducted by GeneCentric scientists in collaboration with researchers at the University of North Carolina Chapel Hill’s Lineberger Cancer Center, is to assess the genomic characteristics of MIBC patients treated with ICPs and to assess the potential implications of those characteristics on treatment responses and outcomes. The study involves applying the Company’s 60-gene 4-class MIBC expression subtyper (BCSP) and an initial FGFR3 activation response signature, developed from MIBC TCGA (The Cancer Genome Atlas) data along with other proprietary assays, to a cohort of de-identified MIBC patients who underwent prior ICP. To stratify and identify positive or negative ICP response indicators, comprehensive genomic analyses were performed on archived FFPE samples from patients within this cohort, followed by tumor and immune profiling as well as subtype and other response associated predictor analyses. The full analysis of the complete dataset (n=97) will be presented at a future meeting.

Details of the presentation are as follows:

Title: "RNAseq and DNA Whole-Exome Sequence Analysis Reveal Novel Response Signatures to IO Treatment in Muscle Invasive Bladder Cancer (MIBC) Patients."

Abstract Number: 4558

Date: June 3, 2019

Time: 1:15 PM to 4:15 PM

Location: Hall A

Presenter: Greg Mayhew, PhD, Director, Bioinformatics, GeneCentric Therapeutics

To read the abstract, please visit: View Source