Seattle Children’s launches BrainChild Bio to accelerate CAR T-cell therapies for children with brain tumors

On December 5, 2023 Seattle Children’s reported the launch of BrainChild Bio, Inc. to accelerate the advancement of chimeric antigen receptor (CAR) T-cell therapies in Central Nervous System (CNS) tumors (Press release, Seattle Children Hospital, DEC 5, 2023, View Source [SID1234638182]). BrainChild Bio will be granted an exclusive license to novel CAR T-cell technology for CNS tumors developed at Seattle Children’s and will build upon the pioneering CAR T-cell therapy and clinical translational work of Michael Jensen, M.D., and his team at Seattle Children’s Therapeutics. Seattle Children’s has provided the initial equity funding for BrainChild Bio which will operate as an independently managed corporation.

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Since 2012, Seattle Children’s Therapeutics has designed, manufactured and launched a robust portfolio of immunotherapy clinical trials for leukemia and lymphoma, brain tumors, and solid tumors, enrolling more than 500 patients. The launch of BrainChild Bio is a natural progression of Seattle Children’s Therapeutics’ goal to expand access to potentially life-changing therapies through collaborations with biotech companies.

"As one of the largest dedicated pediatric institutes in the country, we are extremely proud of the discoveries, clinical trials and cures that have come from inside our own walls," said Dr. Jeff Sperring, Chief Executive Officer of Seattle Children’s. "We also know there are kids around the globe that cannot come to Seattle to be treated. We believe this gives us the best opportunity to accelerate this technology bringing potential cures to kids faster."

BrainChild Bio’s initial CAR T-cell therapy program will focus on pediatric brain tumors, prioritizing diffuse intrinsic pontine glioma (DIPG), an incurable type of childhood cancer that forms in the brainstem. The company’s clinical programs will be accelerated by the foundational work at Seattle Children’s Therapeutics, which consists of four clinical trials designed to validate the safety and confirm early efficacy of several different targets for CAR T-cell therapy in pediatric CNS tumors, with preliminary results planned for presentation at a scientific forum in 2024. The BrainChild-04 clinical study was initiated this year and continues to evaluate four different targets in a single CAR T-cell therapy. Following the achievement of clinical proof-of-concept in DIPG, BrainChild Bio plans to seek pediatric registration for DIPG and then extend the therapeutic application of its novel CAR T-cell therapies to target additional difficult-to-treat pediatric and adult brain tumors, including glioblastoma and brain metastases.

"As a physician, I work with children and their families who have limited therapeutic choices to treat their tumors—and it is devastating that there are few safe and curative options for them," commented Dr. Nicholas Vitanza, CNS CAR T-cell Lead and DIPG Research Lead at Seattle Children’s, and the Founding Chair of Brainchild Bio’s Scientific Advisory Board. "Developing therapies that are tailored specifically for pediatric patients will generate better medicines to address CNS tumors while protecting these young patients and their developing bodies and minds."

BrainChild Bio will optimize the application of CAR T-cell therapies for CNS tumors by advancing a next-generation CAR T-cell therapy platform that integrates synthetic technologies, including multiplex targeting and enhanced potency controls. This multi-dimensional approach includes: (i) multiple targets in a single CAR T-cell therapy to prevent tumor escape; (ii) novel transgenes to increase potency that engage only when within the direct tumor environment; (iii) switching technologies to control the CAR T cells directly within the tumor; (iv) CAR T design and manufacturing processes which have been proven over many years at Seattle Children’s Therapeutics, and (v) novel CAR T-cell administration directly into the brain minimizing systemic toxicities and enabling regular repeat dosing to ensure prolonged presence of CAR T-cells and durable efficacy.

"BrainChild Bio is founded with a mission to bring the best ideas forward to push the bounds of scientific discovery in service of children with cancer. For far too long, children have been deprioritized for commercialized medicines, and families have been left without options," stated Dr. Michael Jensen, Founder and Chief Scientific Officer of BrainChild Bio. "We are steadfast in our commitment to cracking the code of harnessing CAR T-cell technology in CNS tumors and we are uniquely positioned to do so."

BrainChild Bio will be led by world-class business and scientific leaders with a deep track record of innovative drug development. Steven Brugger will serve as Chief Executive Officer, bringing over 40 years of experience leading biopharma companies, most recently as CEO of Affinivax, Inc., a vaccine innovator acquired by GSK for $3.3 billion in 2022. Dr. Michael Jensen will serve as Chief Scientific Officer, directing all research and development efforts focused on commercializing a pipeline of CNS CAR T-cell therapies. Dr. Jensen’s contributions to immunotherapy are significant with over 200 patents in cell and gene therapy, spanning his 30-year career as a physician-scientist, including the last 13 years where he led the R&D team at Seattle Children’s Therapeutics and developed a portfolio of CAR T-cell therapies, directed clinical translation programs and oversaw clinical studies for multiple CAR T-cell therapies. He is the scientific founder of Umoja Biopharma, as well as Juno Therapeutics, which was eventually bought by Bristol Myers Squibb and led to the commercialization of Breyanzi, a CD19-targeting CAR T-cell therapy now commercially available for treating lymphoma in adults.

Odyssey Therapeutics Announces $101 Million Series C Financing

On December 5, 2023 Odyssey Therapeutics, Inc., a biotechnology company pioneering next-generation precision immunomodulators and oncology medicines, reported the closing of a $101 million Series C financing round led by Ascenta Capital with participation from new and existing investors, including OrbiMed, SR One, General Catalyst, Foresite Capital, Woodline Partners LP, HBM Healthcare Investments, Colt Ventures, BlackMars Capital GmbH, Creacion Ventures, funds and accounts advised by Fidelity Management & Research Company, funds and accounts advised by T. Rowe Price Associates, Inc., Catalio Capital Management, Walleye Capital, Alexandria Venture Investments, Racing Beach Ventures LLC, The Healthcare Innovation Investment Fund LLC, an investment fund associated with Leerink Partners, Ab Magnitude Ventures, KB Investment, The Global BioAccess Fund, and multiple leading global investors (Press release, Odyssey HealthCare, DEC 5, 2023, View Source [SID1234638180]). This Series C financing brings the total capital raised since founding in late 2021 to $487 million. Proceeds will support the advancement of multiple programs into clinical studies and the continuation of investment in discovery to build a sustainable model for therapeutic innovation.

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"Odyssey has rapidly advanced a portfolio of immunology and oncology therapeutics with the goal of providing transformative medicines for large numbers of patients in need and several of these molecules have the potential to enter the clinic in the next 12 months," said Gary D. Glick, Ph.D., founder and CEO of Odyssey. "For us, success is defined as bringing safe and effective medicines to patients with serious diseases, with support from Ascenta and our other investors driving our pipeline into the future. We welcome Dr. Lorence Kim, co-founder and managing partner of Ascenta Capital, to our board to join us in achieving this mission."

Dr. Kim brings a wealth of strategic, financial and operational acumen from the biotechnology and pharmaceutical sectors to Odyssey’s board. Notably, during his tenure from 2014 to 2020 as Moderna’s chief financial officer, he raised $4.4 billion to build the company’s technology platform, advance multiple clinical programs and invest in mRNA infrastructure. Prior to Moderna, Dr. Kim advised established pharmaceutical and emerging biotech companies as co-head of biotechnology investment banking at Goldman Sachs.

"Odyssey’s accomplished team of scientists and executives with numerous prior successes in discovery, development and commercialization has made tremendous progress in only two years," said Lorence Kim, M.D. "Dr. Glick has successfully created a sustainable and capital-efficient model that primes Odyssey to be a hub for immunology and oncology therapeutic innovation, and I look forward to working with him, the board and leaders on the management team in the years ahead."

From its headquarters in Boston, Odyssey has built a comprehensive drug discovery and development platform merging both computational and experimental technologies. This unique set of tools enables Odyssey to identify drug targets with the highest clinical potential in a modality-agnostic fashion. In just two years, the company has moved multiple programs forward from ideation to a portfolio of high-value product candidates.

"Odyssey continues to accelerate multiple pre-clinical programs in immunology and oncology," said Jeff Leiden, M.D., Ph.D., Chairman of the Odyssey board. "By raising additional capital from world-class investors and adding a board member with expertise in drug development and company building, we are positioning Odyssey for success over the next several years as our pipeline enters clinical development."

CG Oncology Receives Both FDA Fast Track and Breakthrough Therapy Designation for Cretostimogene Grenadenorepvec in High-Risk BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

On December 5, 2023 CG Oncology, Inc. reported that the U.S. Food and Drug Administration (FDA) has granted both Fast Track Designation and Breakthrough Therapy Designation for cretostimogene grenadenorepvec in high-risk Bacillus Calmette-Guérin (BCG)-unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC) with carcinoma in situ with or without Ta or T1 (papillary) tumors (Press release, CG Oncology, DEC 5, 2023, View Source [SID1234638179]).

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"Receiving both FDA Fast Track and Breakthrough Therapy Designation is an important milestone in the development of cretostimogene grenadenorepvec and for patients with bladder cancer who urgently need more therapeutic options," said Ambaw Bellete, President & Chief Operating Officer, CG Oncology. "We are encouraged by this momentum following our recent announcement of first results from our Phase 3 BOND-003 study for patients with high-risk BCG-unresponsive NMIBC. CG Oncology looks forward to working with the FDA to advance cretostimogene grenadenorepvec as a potential backbone therapy in bladder cancer. We would like to thank the patients, caregivers, investigators and their staff who have participated in the clinical trials."

The FDA decision is informed by the results of CG Oncology’s clinical trials, which showed that patients treated with cretostimogene grenadenorepvec demonstrated clinical benefit based on complete response rates and has been generally well tolerated.

More than 82,000 people are estimated to be diagnosed with bladder cancer this year. NMIBC is the most common form of bladder cancer, representing approximately 75% of newly-diagnosed cases. Four times more men are diagnosed with bladder cancer than women, and it is the sixth most common type of cancer in the United States.

"The Bladder Cancer Advocacy Network (BCAN) is grateful for the expedited review of this potential treatment option for bladder cancer patients with high-risk BCG-unresponsive NMIBC. What patients and their loved ones desperately need are more and better ways to treat their disease," said Andrea Maddox-Smith, Chief Executive Officer of BCAN. "We appreciate the urgency demonstrated by the FDA in recognizing the potential of this therapy."

Cretostimogene grenadenorepvec is in active clinical studies in bladder cancer. Results from an interim analysis of the BOND-003 Phase 3 trial of cretostimogene grenadenorepvec monotherapy for patients with high-risk BCG-unresponsive NMIBC were presented on November 30, 2023, at the 24th Annual Meeting of the Society of Urological Oncology (SUO). The data showed that patients treated with cretostimogene grenadenorepvec had a complete response rate of 75.7% at any time, and was generally well tolerated with no Grade 3 or higher treatment-related adverse events observed. The interim results were based on 66 patients evaluable for efficacy, with an efficacy data cutoff of October 5, 2023.

The FDA’s Fast Track program is intended to facilitate the development and expedite the review of new drugs and biologics designed to treat serious conditions with unmet medical needs. The FDA’s Breakthrough Therapy designation is a process designed to expedite the development and regulatory review of drugs or biologics that are intended to treat serious conditions where preliminary clinical evidence indicating that the drug or biologic may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy.

About Cretostimogene Grenadenorepvec

Cretostimogene grenadenorepvec is an investigational, intravesically delivered oncolytic immunotherapy being evaluated in BOND-003, a Phase 3 clinical trial for the treatment of BCG-unresponsive Non-Muscle Invasive Bladder Cancer. Cretostimogene grenadenorepvec is also being evaluated in a Phase 2 clinical trial (CORE-001) in combination with pembrolizumab in the same indication. In addition, cretostimogene grenadenorepvec is being evaluated in an investigator-sponsored clinical trial in combination with nivolumab for the treatment of muscle invasive bladder cancer.

About the BOND-003 Clinical Study

BOND-003 (NCT04452591) is a single-arm, Phase 3, monotherapy study evaluating cretostimogene grenadenorepvec in patients with high-risk NMIBC unresponsive to BCG. The primary endpoint of the study is complete response (CR) at any time. In a preplanned preliminary analysis, it was reported that patients had a CR rate of 75.7% at any time with no Grade 3 or higher treatment-related adverse events observed. The most common treatment-related adverse events reported include transient grade 1-2 local genitourinary symptoms. No grade 3 or higher adverse events related to cretostimogene grenadenorepvec were observed.

About Bladder Cancer

More than 82,000 people are estimated to be diagnosed with bladder cancer in 2023. NMIBC is the most common form of bladder cancer, representing approximately 75% of newly diagnosed cases. Bladder cancer is the sixth most common form of cancer in the United States, and men account for three quarters of newly diagnosed cases.

Appia Bio to Present Data on API-192 at the 65th American Society for Hematology Annual Meeting and Exposition

On December 5, 2023 Appia Bio, Inc., a biotechnology company developing allogeneic chimeric antigen receptor (CAR)-engineered invariant natural killer T (CAR-NKT) cell therapies from hematopoietic stem cells (HSCs) for patients with cancer, reported that it will present preclinical data on API-192, a first-in-class CAR-NKT allogeneic cell therapy for the treatment of a broad array of B-cell mediated malignancies including non-Hodgkin lymphoma, one of the most common cancers in the U.S (Press release, Appia Bio, DEC 5, 2023, View Source [SID1234638178]).

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These data will be delivered in a poster presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 9-12, 2023 in San Diego, CA.

Title: API-192, an Allogeneic CD19/20 CAR-NKT Product Derived from Cord Blood CD34+ HSPCs for the Treatment of B-cell Malignancies
Date: Monday, December 11, 2023, 6:00-8:00 PM PT
Number: 4801
Session: 703. Cellular Immunotherapies: Basic and Translational: Poster III
Location: San Diego Convention Center Halls G-H
Presenter: Lanny Gov, Ph.D., principal scientist, Appia Bio

API-192 is the first development candidate out of a 2021 collaboration and license agreement between Appia Bio and Kite, a Gilead Company. Under the terms of the agreement, Appia Bio is responsible for preclinical and early clinical research of HSC-derived CAR-NKT product candidates engineered with CARs provided by Kite. Kite is responsible for the later development, manufacturing, and commercialization of the product candidates identified through the collaboration. API-192 is currently undergoing investigational new drug (IND)-enabling studies.

"It has been a fantastic team effort to bring API-192 forward as our first development candidate out of our ACUA technology platform. Our research has benefited from the collaboration and support of our partner, Kite-Gilead, and the opportunity to benchmark to the gold standard set by their autologous cell therapy leadership," said JJ Kang, Ph.D., co-founder and chief executive officer of Appia Bio. "We are excited to share these data that support API-192 as a healthy donor derived next-generation allogeneic cell therapy in lymphoma and bring it to patients in the clinic."

About API-192
API-192 is a human cord blood CD34+ hematopoietic stem and progenitor cell-derived natural killer T (NKT) cell therapy product engineered to express dual chimeric antigen receptors (CARs) targeting two prevalent B-cell antigens, CD19 and CD20 and armored with soluble IL-15 for improved expansion and persistence. Using its proprietary Appia Cells Utilized for Allogeneic (ACUA) platform, Appia Bio can manufacture allogeneic API-192 cells resembling endogenous NKT cells from hematopoietic stem and progenitor cells (HSPCs) at high yield and with high purity without genetic editing or purification steps.

Adcentrx Announces Extension of Series A+ Financing to $51 Million

On December 5, 2023 Adcentrx Therapeutics ("Adcentrx"), a biotechnology company dedicated to revolutionizing Antibody-Drug Conjugate (ADC) therapeutics for cancer and other life-threatening diseases, reported the extension of its Series A+ financing (Press release, Adcentrx Therapeutics, DEC 5, 2023, View Source [SID1234638177]). The additional $13 million adds to the $38 million Series A+ round that was closed in April 2023. The Series A+ extension included participation from Quan Capital and Partners Investment.

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"We are pleased to announce the extension of our Series A+ financing round," said Hui Li, Ph.D., Founder and CEO of Adcentrx. "With the backing of our new investors, we are better positioned to accelerate the clinical development of our lead program, ADRX-0706, and progression of additional pipeline programs towards the clinic. This allows us to continue our mission of creating differentiated therapeutics to address cancer and other life-threatening diseases."

Lighthouse Capital acted as Adcentrx’s financial advisor. The additional proceeds will be used by Adcentrx to continue advancing its propriety ADC therapeutic pipeline in the clinic.

About ADRX-0706
ADRX-0706 is an ADC product candidate discovered by Adcentrx. The antibody component targets Nectin-4, a cell surface adhesion protein over-expressed in multiple human cancers and associated with poor disease prognosis. The ADC is produced using Adcentrx’s proprietary i-Conjugation technology and novel tubulin inhibitor payload, AP052, to generate an ADC with a drug-antibody ratio of eight (DAR 8). ADRX-0706 has a favorable pharmacokinetic and safety profile in preclinical models, and has demonstrated significant efficacy across a variety of tumor indications. ADRX-0706 is currently being evaluated in a Phase 1a/b clinical trial. For more information about the trial, please refer to the Study ID NCT06036121 on ClinicalTrials.gov.