RAPT Therapeutics Announces Positive Data, Including Objective Response Rates and Progression-Free Survival, from its Phase 2 Trial of FLX475 in Combination with a Checkpoint Inhibitor in Patients with Advanced NSCLC

On November 3, 2023 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based therapeutics company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology, reported safety and efficacy data from its Phase 2 trial of FLX475 (tivumecirnon) in patients with advanced non-small cell lung cancer (NSCLC) who had no prior checkpoint inhibitor therapy (Press release, RAPT Therapeutics, NOV 3, 2023, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-announces-positive-data-including-objective [SID1234636896]). The trial evaluated FLX475, an oral small molecule CCR4 antagonist designed to block the migration of regulatory T cells, in combination with the checkpoint inhibitor pembrolizumab. In this cohort of NSCLC patients, 36 patients were evaluable for efficacy, of which 20 were PD-L1 positive. In these PD-L1 positive patients, the combination of FLX475 and pembrolizumab showed a 40% (8/20) confirmed ORR and a median PFS of 6.3 months as of the data cut off date, with seven patients continuing on study. For comparison, historical pembrolizumab monotherapy activity in checkpoint inhibitor-naïve and previously-treated NSCLC patients showed a confirmed ORR of 18% and a median PFS of 4.0 months. The confirmed ORR for the combination of FLX475 and pembrolizumab in PD-L1 low and high subsets were 38% (6/16) and 50% (2/4), respectively. For comparison, the ORR for pembrolizumab monotherapy in the PD-L1 low and high subsets has been previously reported as 10% and 30%, respectively.

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

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

                  Schedule Your 30 min Free Demo!

The data were presented today in a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 38th Annual Meeting being held in San Diego. The presenting author was Julie Brahmer, M.D., Co-director of the Upper Aerodigestive Department and Professor of Oncology, Johns Hopkins University.

"We are excited by the response rates and PFS data for FLX475 in combination with a checkpoint inhibitor in this cohort of NSCLC patients. The data from this cohort, which will continue to mature and potentially improve, met our criteria to advance development of FLX475," said Brian Wong, M.D., Ph.D., President and Chief Executive Officer of RAPT. "We are particularly intrigued to see differentiating efficacy in patients with cool (PD-L1 low) tumors, which are typically poorly responsive to checkpoint inhibitors and checkpoint inhibitor combinations, such as those with anti-TIGIT antibodies. Along with RPT193, we now have two internally discovered compounds that have demonstrated clinical proof of concept in large, commercially attractive indications."

Phase 2 Data Summary in CPI-naïve NSCLC Patients (n=20)

PD-L1 Status Confirmed Responses (n) Confirmed ORR
Positive (TPS ≥1%) 8/20 40%
Low (TPS 1-49%) 6/16 38%
High (TPS ≥50%) 2/4 50%
At the time of data cut off, there was one additional response among the PD-L1 low patients awaiting confirmation.

The combination of FLX475 and pembrolizumab was well tolerated in this Phase 2 NSCLC cohort. The most common treatment-emergent adverse event deemed related to study treatment was QT prolongation that was asymptomatic and reversible. FLX475 has now been dosed in more than 300 patients with various advanced cancers and has been generally well tolerated, and the combination with pembrolizumab has not increased immune-related toxicity beyond that expected with pembrolizumab alone.

In previous disclosures, FLX475 showed durable objective responses as monotherapy in an EBV+ lymphoma, as well as in combination with pembrolizumab in EBV+ gastric cancer.

Webcast Conference Call Information
RAPT will host a webcast conference call today, November 3, 2023 at 10:00 a.m. PT. To join the conference call via phone and participate in the live Q&A session, please pre-register online here to receive a telephone number and unique passcode required to enter the call. The live webcast and audio archive of the presentation may be accessed on the RAPT Therapeutics website at https://investors.rapt.com/events-and-presentations.

About FLX475
FLX475 (tivumecirnon) is a small molecule CCR4 antagonist designed to block the migration of regulatory T cells (Treg) specifically into tumors, but not healthy tissues. Treg represent a dominant pathway for downregulating the immune response, generally correlate with poor clinical outcomes, and may limit the effectiveness of currently available therapies such as checkpoint inhibitors. FLX475 may restore naturally occurring antitumor immunity alone and may synergize with a variety of both conventional and immune-based therapies, such as radiation, chemotherapy, checkpoint inhibitors, immune stimulators, cancer vaccines, and adoptive T cell therapy.

Protara Therapeutics Announces Third Quarter 2023 Financial Results and Business Update

On November 3, 2023 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the third quarter ended September 30, 2023 and provided a business update (Press release, Protara Therapeutics, NOV 3, 2023, View Source [SID1234636895]).

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

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

                  Schedule Your 30 min Free Demo!

"Progress continues across our TARA-002 clinical development programs, with patient dosing now underway in the ADVANCED-2 trial in patients with non-muscle invasive bladder cancer (NMIBC) and the STARBORN-1 trial in pediatric patients with lymphatic malformations (LMs)," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We remain keenly focused on execution across all our ongoing clinical studies and remain on track to report preliminary results from the expansion portion of the ADVANCED-1 trial of TARA-002 in patients with NMIBC in the first half of 2024. With a cash runway into the second quarter of 2025, we believe we are well positioned to achieve key milestones in our TARA-002 development programs."

Recent Highlights

TARA-002 in NMIBC

In September 2023, the Company announced dosing of the first patient in ADVANCED-2, a Phase 2 open-label trial evaluating intravesical TARA-002 in up to 102 NMIBC patients with carcinoma in situ (CIS) (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-naïve (n=27) and BCG-unresponsive (n=75). Trial subjects will receive an induction with or without a reinduction course of six weekly intravesical instillations of TARA-002, followed by a maintenance course of three weekly installations every three months in the BCG-unresponsive cohort.

As previously announced, patient dosing is underway in the Phase 1b ADVANCED-1EXP study, an open-label expansion trial evaluating intravesical TARA-002 at the 40KE1 dose in up to 12 CIS patients, including BCG-naïve, BCG-unresponsive, and BCG-inadequately treated patients. The Company remains on track to report preliminary results in the first half of 2024. The primary endpoint of the trial is the complete response rate at three months.
TARA-002 in LMs

In October 2023, the Company announced dosing of the first patient in STARBORN-1, a Phase 2 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed-cystic LMs. Including an age de-escalation safety lead-in, the trial will enroll approximately 30 patients who will receive up to four injections of TARA-002 spaced approximately six weeks apart. The primary endpoint of the trial is the proportion of participants with macrocystic and mixed cystic LMs who demonstrate clinical success, defined as having either a complete response (90% to 100% reduction from baseline in total LM volume) or substantial response (60% to less than 90% reduction in total LM volume) as measured by axial imaging.
IV Choline Chloride Program

The Company continues to engage with the U.S. Food and Drug Administration and plans to use both regulatory feedback and results from its prevalence study to inform next steps for the IV Choline Chloride development program.
Third Quarter 2023 Financial Results

As of September 30, 2023, cash, cash equivalents and investments were $74.0 million. The Company expects its current cash, cash equivalents and investments will be sufficient to fund its planned operations into the second quarter of 2025.
Research and development expenses for the third quarter of 2023 increased to $6.2 million from $3.5 million during the third quarter of 2022, primarily reflecting an increase in expenses related to non-clinical and clinical trial activities for TARA-002 of $2.2 million as well as $0.4 million of increased employee expenses inclusive of stock-based compensation.
General and administrative expenses for both the third quarter of 2023 and 2022 were $4.5 million.
For the third quarter of 2023, Protara reported a net loss of $9.9 million, or $0.87 per share, compared with a net loss of $7.7 million, or $0.68 per share, for the same period in 2022. Net loss for the third quarter of 2023 included approximately $1.4 million of stock-based compensation expenses.
About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations

Lymphatic malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN). Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. IV Choline Chloride has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PN patients.

About TARA-002 in LMs

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and approved in Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes, and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, and tumor necrosis factor (TNF)-alpha are secreted by immune cells to induce a strong inflammatory reaction and destroy the abnormal cells.

Prime Medicine Reports Third Quarter 2023 Financial Results and Provides Business Updates

On November 3, 2023 Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, reported financial results and provided business updates for the third quarter ended September 30, 2023 (Press release, Prime Medicine, NOV 3, 2023, View Source [SID1234636893]).

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

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

                  Schedule Your 30 min Free Demo!

"In recent weeks, we announced compelling preclinical data across our pipeline, including our first in vivo demonstration of Prime Editing in non-human primates. The consistency of these data provides proof-of-concept that our technology can precisely and efficiently edit across a range of target tissues, correcting pathogenic mutations at levels that may be sufficient to reverse disease manifestations across severe genetic conditions," said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. "We also shared presentations detailing the power of our proprietary delivery systems, as well as new off-target analyses demonstrating the potentially best-in-class safety profile of Prime Editors. Together, these encouraging results mark meaningful progress toward the clinic and support our strategy of simultaneously advancing multiple programs forward. We look forward to leveraging the modularity of our platform, as well as our many clinical, manufacturing and regulatory learnings, to inform further development of our pipeline with the goal of delivering one-time, curative therapies to transform the lives of patients with a wide spectrum of debilitating diseases."
Recent Business Updates

Prime Medicine is advancing a strategic pipeline of eighteen programs. The Company is initially focused on indications with the opportunity for the fastest, most direct path to the clinic and technical success in humans, as well as indications that cannot be treated using other gene editing approaches.

Chronic Granulomatous Disease (CGD)

•In August 2023, Prime Medicine received Rare Pediatric Drug designation (RPDD) from the U.S. Food and Drug Administration (FDA) for PM359. The FDA grants RPDD to medicines intended for the treatment of serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. Companies that receive approval for a New Drug Application or Biologics License Application for a rare pediatric disease may be eligible to receive a voucher for priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the Company or sold to a third party.
Glycogen Storage Disease 1b (GSD1b)

•In October 2023, Prime Medicine presented new in vivo data at the European Society of Gene and Cell Therapy 2023 Congress, demonstrating the ability of Prime Editors to precisely correct one of the most prevalent disease-causing mutations of GSD1b in both non-human primate (NHP) and mouse models. In these preclinical studies using single lipid nanoparticle (LNP) administration, Prime Editors showed up to 50% whole liver precise editing in NHPs (resulting in up to 83% of the key target cells, liver hepatocytes, edited), with no observed safety concerns. In addition, no detectable off-target effects were observed following a comprehensive in vitro off-target screening analysis. These data, the first evidence of Prime Editing in NHPs, provide proof-of-concept for Prime Medicine’s LNP liver-targeted delivery approach and support the further advancement of the Company’s Prime Editors for liver-targeted programs. Read the full data here.
Retinitis Pigmentosa / Rhodopsin
•In October 2023, Prime Medicine presented new in vivo data at the International Symposium on Retinal Degeneration, demonstrating that Prime Editors can efficiently and precisely correct the predominant mutations that cause rhodopsin associated autosomal dominant retinitis pigmentosa (RHO adRP). In preclinical studies, single Prime Editors showed up to 70% precise correction in photoreceptors, as well as favorable tolerability. These data suggest that Prime Medicine’s proprietary dual adeno-associated virus platform can effectively deliver Prime Editors to the eye, with the potential to treat a range of retinal diseases. Read the full data here.
Additional Pipeline and Prime Editing Platform Updates
•Prime Medicine presented additional preclinical data across its Prime Editing pipeline and technology at several healthcare industry conferences in October and has plans to share additional abstracts at medical meetings before year-end. Highlights of the conference presentations included:
◦An overview of Prime Medicine’s comprehensive suite of assays to identify potential off-target events, which has been expanded to include new, unbiased genome-wide tools. These analyses continue to demonstrate minimal to no detectable off-target edits, chromosomal rearrangements or translocations. These preliminary analyses, across multiple editing programs, suggest potentially best-in-class safety;
◦The first presentation on Prime Medicine’s universal LNP-RNA platform designed to deliver Prime Editors to correct pathogenic mutations in the liver;
◦An update on using Prime Editors for the potential treatment of repeat expansion diseases, including an update on Fragile X syndrome; and
◦Initial proof-of-concept data for Prime Medicine’s hotspot approach to developing Prime Editors to correct the CFTR gene in patients with Cystic Fibrosis, as well as new detail on Prime Medicine’s LNP and all-RNA delivery approaches for the lung.
•In December, Prime Medicine will present preclinical data showcasing the potential of the PASSIGE platform to multiplex edit CAR-T cells at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 9-12, 2023, in San Diego, CA. Details of the poster presentation are as follows:
Abstract Title: Multiplex Prime Editing and PASSIGE for Non-Viral Generation of an Allogeneic CAR-T Cell Product
Date & Time: Monday, December 11, 6-8 PM PT
Room: San Diego Convention Center, Halls G-H
Session Title: Cellular Immunotherapies: Basic and Translational
Presenter: Emily Pomeroy
Corporate
•In October 2023, the U.S. Patent and Trademark Office issued Prime Medicine’s third patent, No. 11,795,452, "Methods and Compositions for Editing Nucleotide Sequences," which covers Prime Editing systems that include a PEgRNA, Prime Editor protein, and, optionally, a recombinase.

Anticipated Upcoming Milestones

Year-to-date, Prime Medicine continues to make significant progress, executing against key initiatives to drive its Prime Editing platform forward. The Company initiated IND-enabling studies for PM359 in CGD, and expanded preclinical proof-of-concept data in vivo and in vitro across several programs and target tissue types. In addition, as evidenced by in vivo data shared in recent presentations, Prime Medicine continues to optimize its non-viral and viral delivery systems, and to demonstrate excellent off-target profiles for its Prime Editing programs.
Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward in the coming months:
Pipeline
•Complete first IND filing as early as 2024 with additional IND filings anticipated in 2025.
Platform
•Expand Prime Editing using proprietary recombinase technologies for new and existing programs.
•Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.
Third Quarter 2023 Financial Results:
•R&D Expenses: Research and development (R&D) expenses were $41.0 million for the three months ended September 30, 2023, as compared to $25.0 million for the three months ended September 30, 2022. This increase was primarily due to increases in lab supplies, personnel, and facilities costs as the Company continues to expand and build out its R&D activities and function.

•G&A Expenses: General and administrative (G&A) expenses were $10.5 million for the three months ended September 30, 2023, as compared to $6.6 million for the three months ended September 30, 2022. This increase was primarily due to an increase in professional and consultant costs and personnel costs primarily attributable to the build-out of the Company’s G&A team to support its R&D function.
•Net Loss: Net loss was $50.7 million for the three months ended September 30, 2023, as compared to $29.4 million for the three months ended September 30, 2022.
•Cash Position: As of September 30, 2023, cash, cash equivalents, investments and restricted cash were $178.8 million, as compared to $307.4 million as of December 31, 2022.

Financial Guidance

Based on its current operating plans, Prime Medicine expects that its cash, cash equivalents and investments as of September 30, 2023, will be sufficient to fund its operating expenses and capital expenditure requirements through the end of 2024.

Panbela Announces Exercise of Warrants and Issuance of New Warrants in a Private Placement for $1.9M Gross Proceeds Priced At-the-Market

On November 3, 2023 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that it has entered into agreements with certain holders of its existing warrants exercisable for 2,130,000 shares of its common stock, in the aggregate, to exercise their warrants at a reduced exercise price of $0.78 per share, in exchange for new warrants as described below (Press release, Panbela Therapeutics, NOV 3, 2023, View Source;utm_medium=rss&utm_campaign=panbela-announces-exercise-of-warrants-in-a-private-placement [SID1234636891]). The aggregate gross proceeds from the exercise of the existing warrants is expected to total approximately $1.9 million, before deducting financial advisory fees. The reduction in the exercise price of the existing warrants and the issuance of the new warrants was structured as an at-market transaction under Nasdaq rules.

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

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

                  Schedule Your 30 min Free Demo!

Roth Capital Partners is acting as the company’s financial advisor for this transaction.

The shares of common stock issuable upon exercise of the existing warrants are registered pursuant to a registration statement on Form S-1 (File No.333-271729) which was declared effective by the Securities and Exchange Commission ("SEC") on June 15, 2023.

In consideration for the immediate exercise of the existing warrants for cash and the payment of $0.125 per share underlying the existing warrants, the exercising holders will receive new warrants to purchase shares of common stock in a private placement pursuant to Section 4(a)(2) of the Securities Act of 1933, as amended (the "1933 Act"). Subject to the receipt of stockholder approval for the issuance of the underlying shares of common stock, the new warrants will be exercisable into an aggregate of up to 4,260,000 shares of common stock, at an exercise price of $0.78 per share and have a term of exercise equal to five years after stockholder approval. The new warrants and underlying shares of common stock have not been registered under the Securities Act of 1933, as amended, or applicable state securities laws. Accordingly, the securities may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. As part of the transaction, the company has agreed to file a resale registration statement with the SEC to register the resale of the shares of common stock underlying the new warrants.

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

OncoNano Medicine Announces Positive Preclinical Data for ON-BOARD™ IL-12 Program at SITC 2023 Annual Meeting

On November 3, 2023 OncoNano Medicine, Inc. reported positive preclinical data describing the effective delivery of interleukin-12 (IL-12) with the ON-BOARD platform technology at the 38th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (Press release, OncoNano Medicine, NOV 3, 2023, View Source [SID1234636890]).

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

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

                  Schedule Your 30 min Free Demo!

The study findings show that encapsulation of a therapeutic IL-12 fusion protein (IL-12Fc) for tumor specific delivery and pH-dependent activation was achieved using the ON-BOARD polymeric micelle technology. The study also found that ON-BOARD significantly improved the tolerability of IL-12Fc, as compared to unencapsulated IL-12. The encapsulated IL-12Fc demonstrated potent anti-tumor efficacy and durable anti-tumor memory in the MC38 colorectal cancer model. ONM-412, the development product candidate with the IL-12 encapsulated in the ON-BOARD polymeric micelle system, is advancing through IND-enabling activities.

"These findings underscore the potential application of the ON-BOARD polymeric micelle system for the delivery of IL-12 as an anti-tumor agent," said Tian Zhao, PhD, Vice President of Research and Development at OncoNano. "We look forward to the continued development of ONM-412 and the ONBOARD platform as a potential technology for the targeted delivery of therapeutics to benefit cancer patients."

The data presented demonstrated that:

ON-BOARD enables murine and human IL-12Fc encapsulation with favorable stability and pH-responsive IL-12 bioactivity in vitro in a HEK reporter assay and in primary cell IFNγ induction. Compared to a protease cleavable masked IL-12, the ON-BOARD formulation shows rapid and complete activation over a short period of time.
ON-BOARD encapsulated IL-12Fc exhibited potent anti-tumor efficacy and prolonged survival in MC38 tumor-bearing animals with long-term memory effect.
ON-BOARD formulation demonstrated effective IL-12 signaling in the tumor as evidenced by activation of CD8 T cells and NK cells; increased levels of IFNγ were seen in the tumor but not systemic circulation.
Reduced systemic exposure was observed with ON-BOARD encapsulated IL-12Fc with a significant improvement in the tolerability as evidenced by reduced body weight loss, normal plasma transaminase levels for liver function, reduced splenomegaly, and reduced systemic free IL-12 levels and IL-12 related cytokines when compared to unencapsulated IL-12Fc.

Presentation Details

TITLE: Encapsulation of IL-12 with an ultra pH-sensitive tumor delivery platform improves tolerability and promotes antitumor response in a preclinical model
PRESENTER: Jason Miller, Ph.D., Associate Director, Research Pipeline Development, OncoNano Medicine
POSTER NUMBER: 1147-B