Roche Presents Data at American Society of Hematology (ASH) Annual Meeting

On December 12, 2022 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that Roche issued a press release on data presentation regarding therapeutic antibodies created by Chugai, Hemlibra and crovalimab, at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Chugai, DEC 12, 2022, View Source [SID1234625097]).

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Please refer to the link below for details:

Interim data from phase III study presented at ASH (Free ASH Whitepaper) 2022 show Hemlibra achieved meaningful bleed control in infants from birth
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Roche’s subcutaneous crovalimab given every four weeks achieves disease control in people with PNH, a life-threatening blood condition
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Caribou Biosciences Selects ROR1 as the Target for CB-020, an iPSC-derived Allogeneic CAR-NK Cell Therapy

On December 12, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported target selection for CB-020, an induced pluripotent stem cell (iPSC)-derived allogeneic anti-ROR 1 (receptor tyrosine kinase like orphan receptor 1) CAR-NK cell therapy (Press release, Caribou Biosciences, DEC 12, 2022, View Source [SID1234625093]). Preclinical data on the selection of the CB-020 CAR construct and armoring strategies for Caribou’s CAR-NK cell platform will be presented today at the 12th American Association for Cancer Research (AACR) (Free AACR Whitepaper) and Japanese Cancer Association (AACR-JCA) Joint Conference.

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"ROR1 has been selected as the target for CB-020, Caribou’s first off-the-shelf iPSC-derived CAR-NK cell therapy, and the preclinical data presented at AACR (Free AACR Whitepaper)-JCA shows that ROR1 may be a promising target for several solid tumor indications," said Steve Kanner, Ph.D., Caribou’s chief scientific officer. "We are leveraging our chRDNA genome-editing technology across our allogeneic CAR-T and CAR-NK cell programs to address disease-specific challenges. For solid tumors, we are exploring several armoring strategies for our allogeneic CAR-NK cell therapy platform, including a CBLB knockout, a B2M knockout with a B2M–HLA-E fusion protein insertion, and a membrane-bound IL-15 insertion/IL-15RA fusion protein to help overcome the complex tumor microenvironment that has challenged previous cell therapies."

iPSC-derived NK cells innately exhibit potent antitumor activity against solid tumors. CB-020 is being engineered using Caribou’s Cas12a chRDNA genome-editing technology to express a ROR1-specific CAR, which can enhance the innate NK cell antitumor activity by increasing specificity and function. ROR1 is a cell signaling receptor that is overexpressed on the surface of several solid tumor types and has been shown to drive tumor cell growth, survival, and metastasis. Preclinical data to be presented at AACR (Free AACR Whitepaper)-JCA show that a single dose of iPSC-derived anti-ROR1 CAR-NK cells, administered in a tumor xenograft model, significantly reduced tumor burden compared to iPSC-derived NK cells without an anti-ROR1 CAR.

Multiple armoring strategies are being developed for Caribou’s CAR-NK cell platform to enhance tumor targeting, allogeneic CAR-NK cell survival, and persistence of antitumor activity. Results from the company’s preclinical studies suggest iPSC-derived NK cells with a knockout of CBLB (Casitas B-Lineage lymphoma proto-oncogene-B), a ubiquitin ligase that negatively regulates NK cell function, results in reduced tumor burden and increased overall survival in an in vivo solid tumor xenograft model, compared to unedited iPSC-derived NK cells. Additionally, results show that iPSC-derived NK cells were not killed by donor-derived T cells and NK cells when harboring a knockout of B2M and an insertion of a BM2–HLA-E fusion protein. This strategy may induce more potent NK activity and help prevent CAR-NK cells from killing each other, which is a common problem with NK cell therapies. In addition, results from iPSC-derived NK cells with an insertion of membrane-bound IL-15/IL-15RA

fusion protein, which is shown to enhance NK cell antitumor activity, demonstrated high cytotoxicity against tumor cells compared to unedited iPSC-derived NK cells. Together, these preclinical data demonstrate Caribou’s genome-editing technology has the potential to be used to implement a variety of armoring strategies in iPSC-derived CAR-NK cells to address many of the challenges associated with treating solid tumors.

Details of the poster presentation at the AACR (Free AACR Whitepaper)-JCA Joint Conference are as follows:

Title: CB-020, an iPSC-derived allogeneic CAR-NK cell therapy
Speaker: Rudy Gonzalez, Ph.D., executive director of stem cell therapeutics, Caribou Biosciences, Berkeley, CA
Date and time: Monday, December 12 at 5:30 pm HST
Abstract number: B06
Location: Hyatt Regency Maui, Monarchy Ballroom

The full poster is available on Caribou’s Scientific Publications (www.cariboubio.com/technology/#pubs) webpage.

PharmaMar receives US$10 million milestone payment from Janssen

On December 12, 2022 PharmaMar (MSE:PHM) has reported receipt of a payment of $10 million from Janssen Products LP after reaching a commercial milestone for Yondelis (trabectedin) in the United States as defined in the licensing agreement (Press release, PharmaMar, DEC 12, 2022, View Source [SID1234625092]).

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In August 2019, PharmaMar entered into a new licensing agreement with Janssen, which replaced a 2001 licensing agreement, under which Janssen retained the right to sell and distribute, on an exclusive basis, Yondelis in the USA.

Today, Yondelis is approved in more than 70 countries for the treatment of soft tissue sarcoma and in some of these countries ovarian cancer as well.

Caribou Biosciences Reports CB-010 ANTLER Phase 1 Trial Progress

On December 12, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported new 12-month clinical data from cohort 1 in the ongoing ANTLER Phase 1 trial, which show long-term durability following a single infusion of CB-010 at the initial dose level 1 (40×106 CAR-T cells) (Filing, 8-K, Caribou Biosciences, DEC 12, 2022, View Source [SID1234625090]). Cohort 1 results show:

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•6 of 6 patients achieved a complete response (CR) as best response

•3 of 6 patients maintained a durable CR at 6 months

•2 of 6 patients maintain a long-term CR at the 12-month scan and remain on the trial

•18 months is the longest CR maintained to date in ANTLER, achieved by the first patient dosed with CB-010

•CB-010 was generally well tolerated with adverse events consistent with autologous or allogeneic anti-CD19 CAR-T cell therapies

Based on promising initial data, the U.S. Food and Drug Administration (FDA) granted CB-010 both Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations. In addition, Caribou has observed an encouraging safety profile for CB-010 at dose level 2 (80×106 CAR-T cells) with no dose-limiting toxicities (DLTs) in the 3 patients treated and is currently enrolling patients at dose level 3 (120×106 CAR-T cells). Caribou expects to provide an ANTLER trial update in 2023.

"With next-generation CRISPR genome-editing technology, the promise of allogeneic cell therapies has advanced significantly, and the early results seen in the ANTLER trial to date are a reflection of

that potential," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "The long-term durability at dose level 1 is comparable to autologous cell therapies and we believe CB-010 has the potential to set a new therapeutic bar for what allogeneic anti-CD19 CAR-T cell therapies can achieve. We are further encouraged by receiving RMAT and Fast Track designations for CB-010 from the FDA, which is a testament to both the encouraging initial ANTLER data and the need for novel therapies for patients with relapsed or refractory B-NHL."

CB-010 is the first allogeneic anti-CD19 CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to improve the persistence of antitumor activity by limiting premature CAR-T cell exhaustion.

"Patients with relapsed or refractory B cell non-Hodgkin lymphoma are in need of treatments that are immediately available and do not require burdensome or ineffective bridging therapies," said Susan O’Brien, M.D., professor of medicine, Chao Family Comprehensive Cancer Center at University of California, Irvine, CA, and presenting investigator on the ANTLER clinical trial. "The early results from the ANTLER trial are promising and I look forward to enrolling additional patients in this trial to learn more about the potential of CB-010 as an off-the-shelf treatment option for patients with aggressive B-NHL who have a high unmet medical need."

ANTLER is a Phase 1, open-label, multicenter clinical trial (NCT04637763) evaluating the safety and efficacy of the company’s lead allogeneic cell therapy, CB-010, in patients with r/r B-NHL. The trial includes Part A, a 3+3 dose escalation phase designed to evaluate safety of CB-010 at multiple dose levels and establish the recommended Phase 2 dose, and Part B, a dose expansion phase with the primary objective to determine tumor response after a single dose of CB-010. As permitted by the protocol, backfilling patients has begun at doses deemed well tolerated to increase the understanding of CB-010’s safety profile and antitumor activity and provide additional data for establishing a recommended Phase 2 dose.

ANTLER Trial-in-Progress Poster at ASH (Free ASH Whitepaper) 2022
Today at the 64th Annual ASH (Free ASH Whitepaper) meeting, a trial-in-progress poster is being presented to provide details of the design and objectives of the ANTLER Phase 1 trial for CB-010 in r/r B-NHL. Details of the poster presentation are as follows:

Title: A First-in-Human Phase 1, Multicenter, Open-Label Study of CB-010, a Next-Generation CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy with a PD-1 Knockout, in Patients with Relapsed/Refractory B Cell Non-Hodgkin Lymphoma (ANTLER Study)
Presenter: Susan O’Brien, M.D., professor of medicine, Chao Family Comprehensive Cancer Center at University of California, Irvine, CA
Session Name: 626. Aggressive Lymphomas: Prospective Therapeutic Trials: Poster III
Session Date: Monday, December 12, 2022
Presentation Time: 6:00 pm – 8:00 pm CST
Location: Ernest N. Morial Convention Center, Hall D
Abstract number: 4257

The poster presentation will be available for registered attendees on the ASH (Free ASH Whitepaper) website and on Caribou’s website under Scientific Publications (www.cariboubio.com/technology/#pubs) on Monday, December 12, 2022 at 9:00 am CST.

About CB-010
CB-010 is the lead product candidate from Caribou’s allogeneic CAR-T cell therapy platform and is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) in the ongoing ANTLER Phase 1 trial. CB-010 is an allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9 CRISPR hybrid RNA-DNA (chRDNA) technology to insert a CD19-specific CAR into the TRAC gene and knock out PD-1 to boost the persistence of antitumor activity. CB-010 is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knock out. CB-010 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations. Additional information on the ANTLER trial can be found at www.clinicaltrials.gov using identifier NCT04637763.

8-K – Current report

On December 12, 2022, PDS Biotechnology Corporation reported its corporate presentation deck (Presentation, PDS Biotechnology, DEC 12, 2022, View Source [SID1234625091]). A copy of the corporate presentation is filed as Exhibit 99.1 and incorporated herein by reference.

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