Infinity Reports Clinical and Translational Data from Expansion Cohorts of MARIO?1 Phase 1b Study of IPI-549 in Combination with Opdivo® (nivolumab) at SITC’s 33rd Annual Meeting

On November 10, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported that data to be presented today at the 33rd Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) demonstrated preliminary evidence that IPI-549 in combination with Opdivo (nivolumab) is clinically active in indications not expected to respond to Opdivo alone (Press release, Infinity Pharmaceuticals, NOV 10, 2018, View Source [SID1234531169]). In particular, evidence of reversal of resistance to Opdivo included a partial response in a patient with metastatic melanoma who progressed on immediate prior Opdivo therapy. IPI-549 plus Opdivo also resulted in a 26% reduction of tumor target lesions in a patient with chemotherapy-resistant triple negative breast cancer (TNBC), a tumor type intrinsically resistant to checkpoint inhibition. The data also included long-term follow up on sustained partial responses in two patients from combination dose escalation: one with microsatellite stable (MSS) gallbladder cancer and one with adrenocortical carcinoma. The observed early clinical activity from the combination expansion, in addition to findings from the monotherapy cohorts, including associated translational data, support on-mechanism proof of concept for IPI-549. The late-breaking abstract describing these findings will be presented today in a poster presentation at the SITC (Free SITC Whitepaper)’s 33rd Annual Meeting taking place in Washington, D.C., November 7 – 11, 2018.

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"There continues to be a significant need for improvement in the treatment of patients with advanced solid tumors who do not respond to checkpoint inhibitors alone. IPI-549’s potential to improve upon the existing CPIs makes it an important first-in-class drug in development," said David Hong, M.D. Deputy Chair, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX. "The reversal of resistance to checkpoint inhibition is particularly encouraging for the further development of IPI-549, and I look forward to my continued participation in the development of IPI-549."

Infinity is evaluating IPI-549 in MARIO-1, a Phase 1b study in approximately 200 patients with advanced solid tumors. Additionally, Infinity is planning to initiate MARIO-275, a global, randomized Phase 2 study to evaluate the effect of adding IPI-549 to Opdivo in approximately 150 checkpoint-inhibitor naïve advanced urothelial cancer patients who have progressed or recurred following treatment with platinum-based chemotherapy.

"The rigorous design of our Phase 1b study sets a high bar for the determination of proof of concept, given that the goals of our study are to see clinical activity both in settings where patients are not expected to respond to Opdivo alone, and in indications where there are high levels of immunosuppressive macrophages," said Dr. Sam Agresta, Chief Medical Officer of Infinity Pharmaceuticals. "We do this with the intention of re-sensitizing patients to Opdivo to overcome the limiting effects of macrophages to checkpoint inhibitor therapy, and we are excited that the data we shared today demonstrate clinical activity in this patient population. We look forward to the maturation of MARIO-1 and the advancement of the IPI-549 clinical development program in the checkpoint-inhibitor naïve setting with MARIO-275."

"Today’s data validate our scientific rationale for the development of IPI-549 as a potentially first-in-class therapeutic alternative for patients lacking better treatment options," said Adelene Perkins, Chief Executive Officer of Infinity Pharmaceuticals. "We will be expanding our melanoma cohort, with patients refractory to immediate prior anti-PD1 therapy, to 40 patients based on promising early signals and have triggered expansion of the TNBC cohort. We will also be initiating our Phase 2 MARIO‑275 trial in urothelial cancer with an emphasis on patients with high myeloid derived suppressor cell levels, given that MDSCs further promote immunosuppression. We look forward to providing an update on these efforts."

Details of Today’s Late-Breaking Presentation

Infinity will present a poster entitled "The first clinical/translational data from the expansion cohorts of a Ph1/1b Study of IPI-549, a tumor macrophage-reprogramming small molecule, in combination with nivolumab in advanced solid tumors" (Poster P716). The data reported today from an October 14, 2018 data cutoff included 82 patients treated with IPI-549 at 40 mg QD and Opdivo at 240mg IV once every two weeks.

Summary of Data

Combination expansion data demonstrated that IPI-549 in combination with Opdivo is well tolerated and is associated with a favorable safety profile. Among 82 patients evaluable for safety, the majority of side effects reported were Grade 1 or Grade 2, with 3 (4%) patients discontinuing the study due to treatment-related toxicities. There were no treatment-related deaths. These 82 patients have been treated in seven distinct cohorts: non-small cell lung cancer (n=8), melanoma (n=15), head and neck cancer (n=12), triple negative breast cancer (n=17), adrenocortical cancer (n=5), mesothelioma (n=11), and a biomarker defined baseline MDSC high cohort (n=14). The majority of the study population is 4th line and resistant to anti-PD1/PDL1 therapy. Among the 44 patients evaluable for activity (approximately one-third of the planned 129 patients in the combination expansion cohorts), 15 patients showed a best response of stable disease or better, including one partial response in an advanced melanoma patient who progressed on immediate prior Opdivo therapy. In addition, a patient with chemotherapy-resistant triple negative breast cancer showed 26% reduction in tumor target lesions at the first assessment. Reductions in elevated baseline levels of MDSCs as well as corresponding increases in the proliferative fraction of previously exhausted memory cytotoxic T-cells were seen in these patients. Another partial response was observed in a patient with advanced mesothelioma. Twenty-five patients remain on study and were not evaluable for activity as of the data cutoff. The data included long-term follow up on additional partial responses in two patients from the combination dose escalation: one with microsatellite stable gallbladder cancer and one with adrenocortical carcinoma and these two partial responses were maintained for over 12 and 17 months, respectively. These patients also demonstrated sustained inhibition of MDSCs during the period in which the partial response was maintained. Enrollment is ongoing.

Infinity Investor/Analyst Reception and Webcast
In conjunction with SITC (Free SITC Whitepaper)’s 33rd Annual Meeting, Infinity will host a reception and webcast for investors and analysts today, November 10, 2018, beginning at 6:30 a.m. ET to discuss the clinical development of IPI-549, including a review of data from the Phase 1b clinical study.

The event will feature David Hong, M.D., Deputy Chair, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX.

The webcast and accompanying slides can be accessed in the "investors/media" section of the company’s website, www.infi.com. A replay of the event will also be available.

About IPI-549 and the Ongoing MARIO-1 Phase 1/1b Study
IPI-549 is an investigational first-in-class, oral, immuno-oncology product candidate targeting tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition, thereby reducing pro-tumor macrophage function and increasing anti-tumor macrophage function. In preclinical studies, IPI-549 demonstrated the ability to reprogram macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and enhance the activity of, and overcome resistance to, checkpoint inhibitors.i ii As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially additive or synergistic approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

The ongoing Phase 1/1b study being conducted by Infinity is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with nivolumab (Opdivo) in approximately 200 patients with advanced solid tumors.iii The study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation and expansion components are complete. The combination dose-escalation component is also complete, and combination expansion cohorts are enrolling.

The combination expansion component of the study includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer, melanoma and head and neck cancer whose tumors show initial resistance or initially respond to but subsequently develop resistance to immune checkpoint blockade therapy. The combination expansion component also includes a cohort of patients with triple negative breast cancer who have not been previously treated with immune checkpoint blockade therapy, a cohort of patients with mesothelioma, a cohort of patients with adrenocortical carcinoma and a cohort of patients with high baseline blood levels of MDSCs.

About the Planned MARIO-275 Phase 2 Study
Infinity is planning to conduct MARIO-275: MAcrophage Reprogramming in Immuno-Oncology, a global, randomized Phase 2 study to evaluate the effect of adding IPI-549 to Opdivo in checkpoint-naïve advanced urothelial cancer patients who have progressed or recurred following treatment with platinum-based chemotherapy. Approximately 150 patients will be randomized between combination therapy and Opdivo monotherapy. The primary endpoint of the trial will be overall response rate, which will be assessed in the overall population as well as in subsets of patients with different baseline levels of myeloid derived suppressor cells (MDSCs). Opdivo is approved for use by the FDA as a single agent in patients with locally advanced or metastatic urothelial cancer who have progressed or recurred following treatment with platinum-based chemotherapy or who have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. In exploratory analyses of the CheckMate-275 data, high levels of MDSCs were associated with shorter overall survival in patients treated with Opdivo2. In Infinity’s MARIO-1study, MDSCs were reduced in the majority of patients treated with IPI-549 monotherapy.3 IPI-549 in combination with Opdivo has been administered to over 80 patients and demonstrated early evidence of clinical activity with translational studies demonstrating evidence of on-mechanism IPI-549-mediated effects.4

IPI-549 is an investigational compound, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

Brooklyn ImmunoTherapeutics Announces Presentation of Positive Results of IRX-2 Therapy in Resectable Breast Cancer and Head and Neck Cancer at the 33rd Annual Meeting of the Society for Immunotherapy of Cancer

On November 10, 2018 Brooklyn ImmunoTherapeutics, a biopharmaceutical company focused on exploring the role that cytokine-based therapy can have in treating patients with cancer, and Providence Cancer Institute reported the clinical results of IRX-2 therapy in resectable breast cancer and head and neck cancer that were presented in an oral presentation at the 2018 Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) on November 10th at the Walter E. Washington Convention Center in Washington, D.C (Press release, Brooklyn ImmunoTherapeutics, NOV 10, 2018, View Source [SID1234531112]).

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IRX-2 is an allogeneic, cell-derived biologic with multiple active cytokine components, including IL-2, that act on various parts of the immune system to activate the entire tumor microenvironment.

"The clinical results presented today demonstrate that treatment with IRX-2 was associated with a mean 116% increase in tumor-infiltrating lymphocytes in patients with early stage breast cancer and a mean increase of 58% in head and neck squamous cell carcinoma," said David B. Page, M.D., Earle A. Chiles Research Institute, a division of Providence Cancer Institute in Portland, Oregon, and the presenter at SITC (Free SITC Whitepaper)’s 33rd Annual Meeting. "Moreover, IRX-2 therapy was also associated with an increase in PD-L1 RNA upregulation in the early stage breast cancer patients and was well-tolerated in both the breast cancer and head and neck cancer patients enrolled in these clinical trials. Together these results support the further evaluation of IRX-2 with anti-PD-1 and neoadjuvant chemotherapy in stage II-III triple negative breast cancer as well as ongoing follow-up of a randomized Phase 2 INSPIRE trial in head and neck cancer."

"These highly encouraging clinical results support further study of IRX-2 as a potential important new immunotherapeutic drug candidate for the treatment of both breast cancer and head and neck cancer," said Mark Leuchtenberger, interim President and CEO of Brooklyn ImmunoTherapeutics. "We believe that IRX-2, both as a single agent and in combination with other anti-cancer agents, can potentially improve patient outcomes in these difficult-to-treat indications as well as in the treatment of other cancers. On-going studies, including the Phase 2B INSPIRE trial and an investigator-sponsored trial in squamous cervical intraepithelial neoplasia 3 or vulvar intraepithelial neoplasia 3, are further exploring the potential of IRX-2 in treating cancer."

Results

In the early stage breast cancer trial (ESBC), 16 patients were enrolled and evaluable for tumor-infiltrating lymphocyte (TIL) analysis, and the head and neck squamous cell carcinoma (HNSCC) trial is fully enrolled at 105 patients with 36 patients evaluable at the time of analysis. In both trials, all patients received all planned injections with no treatment-related surgical delays, complications, or treatment-related grade III/IV toxicities. Treatment was associated with a mean 116% relative increase in TILs (range –36% to +1275%, p = 0.02) in ESBC and a mean 58% relative increase (range –57 to +452%, p=0.01) in HNSCC. Treatment was associated with PD-L1 RNA upregulation in EBSC (mean +54%, range –53% to +185%, p=0.04). RNA analysis in ESBC and HNSCC revealed concordant increases in cytokine gene expression, including CXCL2, CCL4, CXCR4, and CXCL12 as well as transcription factors including FOS, ETS1, NFκB, EGR1/2 which are involved in T-cell activation and differentiation. Augmentation of ITGAE (CD103), a known marker of memory T-cell activation in EBSC cohort was also observed

Celldex Presents Emerging MerTK Antibody Program at the Society for Immunotherapy of Cancer’s 33rd Annual Meeting

On November 10, 2018 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported data from the Company’s MerTK antibody program in a poster session at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting(Press release, Celldex Therapeutics, NOV 10, 2018, View Source [SID1234531099]). MerTK is emerging as a promising target for cancer immunotherapy. Its expression in innate immune cells is believed to negatively regulate immune responses and genetic removal of MerTK renders mice resistant to some tumors.

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"MerTK has been described as an immune checkpoint in macrophages, dendritic cells and other immune cells," said Tibor Keler, Ph.D., Executive Vice President and Chief Scientific Officer of Celldex Therapeutics. "Through our significant discovery effort, we have identified two unique antibodies that modulate this pathway resulting in profound levels of cytokine and chemokine production, and importantly we have developed preclinical models that support the premise that antibody modulation of MerTK can lead to antitumor responses."

As detailed in the presentation, Celldex developed a large panel of antibodies to MerTK and investigated their ability to enhance activation of innate immune cells. Two lead candidate human anti-MerTK antibodies were then selected based on their potent induction of cytokines from human macrophages, dendritic cells, and monocytes. Treatment of dendritic cells with the MerTK antibodies led to production of a broad array of pro-inflammatory cytokines and chemokines. Isolated peripheral blood monocytes were found to express high levels of MerTK and were similarly activated by the MerTK antibodies.

Emerging proof of concept data was established in preclinical models. Using a surrogate anti-mouse MerTK​ antibody, similar increases in the levels of cytokines were observed in the blood of mice shortly after treatment with the antibody. The anti-mouse MerTK​ antibody led to increased survival when dosed alone or in combination with a PD-1 inhibitor in a colon cancer model. To test the lead clinical candidates, which bind to human and not mouse MerTK, Celldex generated human MerTK transgenic mice that were shown to appropriately express and regulate human MerTK on macrophages. This will now allow testing of the anti-human MerTK mAbs in inflammation and tumor models. Collectively, the data support that anti-MerTK mAbs can modulate MerTK activity consistent with its role as a negative immune regulator and provide an exciting new approach to enhance innate immune function in cancer.

Celldex is currently completing the preclinical studies for selection of the lead candidate to advance into development activities. These studies include investigating the antitumor effect of combinations with Celldex’s immunotherapy product candidates.

CRISPR Therapeutics and MaxCyte Expand Clinical and Commercial License Agreement into Oncology

On November 9, 2019 CRISPR Therapeutics (NASDAQ:CRSP) and MaxCyte reported the expansion of their existing relationship by entering into a non-exclusive commercial license agreement that will allow CRISPR Therapeutics to deploy MaxCyte’s Flow Electroporation Technology to develop CRISPR/Cas9-based therapies in immuno-oncology (Press release, MaxCyte, NOV 9, 2018, View Source [SID1234537624]).

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"As we advance our allogeneic CAR-T programs into the clinic, we are preparing for the future by securing our access to the leading ex vivo delivery platform for both clinical and commercial use, just as we previously did for our hemoglobinopathy developmental candidates," said Samarth Kulkarni, CEO of CRISPR Therapeutics.

The expanded relationship builds on an existing agreement announced in March 2017 which allowed for the development of commercial therapeutics for hemoglobin-related diseases. Under the terms of the new license agreement, CRISPR Therapeutics will obtain non-exclusive development and commercial-use rights to MaxCyte’s cell engineering platform to develop immuno-oncology cell therapies. MaxCyte will supply its technology to CRISPR Therapeutics as part of the enabling technology license agreement and will receive milestone and sales-based payments in addition to other licensing fees.

"The expansion of our relationship with CRISPR Therapeutics signifies a key milestone for MaxCyte and our technology, providing further validation for the value and versatility of our technology as a leading enabler of next-generation cell-based therapies," said Doug Doerfler, President & CEO of MaxCyte, Inc. "CRISPR Therapeutics is a leader in gene editing, and we are very pleased to expand our collaboration into new therapeutic areas as we continue to explore the use of our technology to advance medicines to market that will make a difference for patients."

MaxCyte’s Flow Electroporation Technology enables the engineering of a broad range of therapeutically-relevant cell types at high efficiency while maintaining high viability and recovery. CRISPR Therapeutics’ immuno-oncology cell therapy programs rely on ex vivo gene editing, where the CRISPR components are delivered into T-cells using the MaxCyte technology.

SITC 2018: further analyses of the Phase II study of the combination of monalizumab and cetuximab in head and neck patients show encouraging survival data in both subgroups of IO-naïve and IO-pretreated SCCHN patients

On November 12, 2018 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH), reported exploratory subgroup analyses and preliminary translational data from the Phase II trial evaluating the combination of monalizumab and cetuximab (anti-EGFR) in previously treated patients with recurrent and/or metastatic squamous cell carcinoma of the head & neck (R/M SCCHN) (Press release, Innate Pharma, NOV 9, 2018, View Source [SID1234531226]). Monalizumab is a first-in-class checkpoint inhibitor targeting NKG2A inhibitory receptors expressed on tumor-infiltrating cytotoxic CD8 T lymphocytes and NK cells.

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"The subgroup analysis revealed very interesting durable responses in both IO-naïve and IO-pretreated patients," commented Pierre Dodion, Chief Medical Officer of Innate Pharma. "Patients who have failed on IO-therapy currently have no other treatment option. The fact that they appear to benefit with prolonged survival is rather remarkable and prompted us to advance our clinical program. Discussions are taking place in parallel with regards to the next steps in IO naïve patients".

"The high disease control rate that we observed in this study along with the favorable trends in progression-free and overall survival, including a preliminary estimate of median overall survival of 10.3 months, are very encouraging," commented Professor Roger B. Cohen, Principal Investigator of the study. "We also saw evidence of clinical activity in both IO-naive and IO-pretreated patients. If confirmed, these data would provide support for a highly novel immunotherapeutic combination with a unique mechanism of action for the treatment of patients with head & neck cancer."

Data from the cohort of 40 patients were reported at 2018 ESMO (Free ESMO Whitepaper) congress in October. Exploratory subgroup analysis revealed encouraging responses, durability of response, PFS and OS in IO-naïve and IO-pretreated patients.

Cut-off August 31, 2018

All patients

(n=40)

IO-naïve

(n=23)

IO-pretreated

(n=17)

ORR [95% CI]

27.5% [16-43]

35% [19-55]

18% [6-41]

Median PFS [95% CI]

5.0 m [3.7-6.9]

4.0 m[3.7-10.6]

5.0 m [3.5-NR]

Median OS [95% CI]

10.3 m [7.3-NR]

10.3 m [7.2-NR]

12.8 m [6.0-NR]

DCR 24 weeks

35% [22-51]

39% [22-59]

29% [13-53]

Median time to response [range]

1.6 m [1.5-3.9]

1.7 m [1.5-3.9]

1.6 m [1.6-3.1]

Median duration of response [95% CI]

5.6 m [3.8-NR]

5.3 m [3.8-NR]

5.6 m [3.7-NR]

Eric Vivier, Chief Scientific Officer of Innate Pharma, said: "We are combining the use of state-of-the-art technologies including high-dimensional flow cytometry, single cell RNA seq and computational pathology, to monitor the immune response in patients. Data support the mode of action of monalizumab as unleashing a multilayered immune response involving both NK and T cells. As such, monalizumab appears as a first-in-class second generation immune checkpoint inhibitor with a large spectrum of immune targets. He added: "While preliminary, translational results suggest that the combination of monalizumab and cetuximab is associated with a reshaping of "cold tumors" into "hot tumors" prone to sustain tumor immunity".

Preliminary biomarker analysis show indeed that while the combination did not impair the distribution or absolute counts of peripheral NKG2A+ NK and CD8+ T cells, an infiltration of NK cells and CD8+T cells is detected very early upon treatment (day 15) at the tumor bed. Further, tumor-infiltrating NK and CD8+ T cells may be predictive of RECIST response, tumor reduction and PFS.

Importantly, the activity of the monalizumab and cetuximab combination appears to occur across HPV status, tumor burden and PD-L1 expression.