On December 22, 2022 Exelixis, Inc. (Nasdaq: EXEL) reported the initiation of STELLAR-304, a phase 3 pivotal trial evaluating zanzalintinib in combination with nivolumab versus sunitinib in patients with advanced non-clear cell renal cell carcinoma (nccRCC) (Press release, Exelixis, DEC 22, 2022, View Source [SID1234625536]). Zanzalintinib, which was adopted as the generic name for XL092, is a next-generation tyrosine kinase inhibitor (TKI) in development for multiple advanced tumor types.

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"In September at ESMO (Free ESMO Whitepaper) 2022, we presented zanzalintinib phase 1 data which demonstrated promising clinical activity across a range of tumors with a manageable safety profile. We were particularly encouraged by the activity of zanzalintinib in advanced kidney cancer patients, including patients with non-clear cell subtypes. Based on this zanzalintinib data and given that nivolumab has shown activity in non-clear cell kidney cancer, we are excited to evaluate this combination regimen in this population in STELLAR-304," said Vicki L. Goodman, M.D., Executive Vice President, Product Development & Medical Affairs, and Chief Medical Officer, Exelixis. "STELLAR-304 is the first and only randomized controlled phase 3 study to focus specifically across non-clear cell renal cell carcinoma subtypes, a patient population with limited clinical data and poorer treatment outcomes. We look forward to continuing our legacy of working towards improving care for all kidney cancer patients."

STELLAR-304 is a global, multicenter, randomized phase 3 open-label study that will enroll approximately 291 patients with unresectable, locally advanced or metastatic nccRCC with no prior systemic anticancer therapy. One prior systemic adjuvant therapy, including immune checkpoint inhibitor (ICI) therapy and excluding sunitinib, is allowed for completely resected RCC and if recurrence occurred at least six months after the last dose of adjuvant therapy. Patients will be randomized 2:1 to receive either zanzalintinib in combination with nivolumab or sunitinib monotherapy. The primary objective of the study is to evaluate the efficacy of the combination, as measured by duration of progression-free survival and objective response rate per Response Evaluation Criteria in Solid Tumors version 1.1 as assessed by the Blinded Independent Radiology Committee. The secondary endpoint is duration of overall survival.

STELLAR-304 is sponsored by Exelixis, and Bristol Myers Squibb is providing nivolumab for the trial.

About Zanzalintinib (XL092)
Zanzalintinib is a next-generation oral TKI that inhibits the activity of receptor tyrosine kinases implicated in cancer growth and spread, including VEGF receptors, MET, AXL and MER. These receptor tyrosine kinases are involved in both normal cellular function and in pathologic processes such as oncogenesis, metastasis, tumor angiogenesis and resistance to multiple therapies, including ICIs. In designing zanzalintinib, Exelixis sought to build upon its extensive experience with the target profile of cabozantinib, the company’s flagship medicine, while improving key characteristics, including pharmacokinetic half-life. Zanzalintinib is currently being developed for the treatment of advanced solid tumors, including genitourinary cancers, as a monotherapy and in combination with ICIs.

About RCC
The American Cancer Society’s 2022 statistics cite kidney cancer as among the top 10 most commonly diagnosed forms of cancer among both men and women in the U.S.1 Clear cell RCC is the most common type of kidney cancer in adults.2 nccRCC represents about 20-25% of RCC cases, with fewer treatment options available and poorer outcomes compared with clear cell RCC.3 If RCC is detected in its early stages, the five-year survival rate is high; for patients with advanced or late-stage metastatic RCC, however, the five-year survival rate is only 14%.1 Approximately 33,000 patients in the U.S. and more than 71,000 worldwide will require systemic treatment for advanced kidney cancer in 2022, with over 15,000 patients in need of a first-line treatment in the U.S.4

On December 22, 2022 Eli Lilly and Company (NYSE: LLY) and ProQR Therapeutics N.V. (Nasdaq: PRQR), reported the expansion of their licensing and collaboration agreement focused on the discovery, development and commercialization of new genetic medicines (Press release, Eli Lilly, DEC 22, 2022, View Source [SID1234625535]).

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The collaboration, originally announced in September 2021, applied ProQR’s proprietary Axiomer RNA editing platform to target disorders of the liver and nervous system. Through the course of work to date, advances in the platform have significantly increased editing efficiency and refined biodistribution in both the liver and nervous system, opening up new potential applications to not only correct known mutations, but also introduce protective variants in specific transcripts. Through this expanded collaboration, Lilly and ProQR will explore further applications of the Axiomer platform to unlock new innovative treatments for people living with diseases with high unmet medical need.

"Discovering and developing the medicines of tomorrow takes time, sustained innovation and most importantly, collaboration," said Andrew C. Adams, Ph.D., Lilly senior vice president of genetic medicine and co-director of the Institute for Genetic Medicine. "We have been impressed with the progress to date with our partners at ProQR and have conviction that RNA editing can be an important alternative to other more permanent therapies."

"Our original collaboration with Lilly, which leverages our Axiomer RNA editing technology platform, continues to progress well and we are pleased to be expanding our partnership to include additional targets, along with an option for Lilly to opt in for more," said Daniel A. de Boer, founder and CEO of ProQR. "Lilly is a leader in RNA therapeutics, and our expanded partnership is another validation of our leadership in ADAR-mediated RNA editing, our robust IP estate, and the potential of our broadly applicable Axiomer platform technology. We look forward to making an impact on the lives of patients together with Lilly."

Under the terms of the expanded agreement, Lilly will gain access to additional targets in the central nervous system and peripheral nervous system with ProQR’s Axiomer platform. ProQR will receive $75 million consisting of an upfront payment, as well as an equity investment. Lilly will have the ability to exercise an option to further expand the partnership for a consideration of $50 million. In addition, Lilly can elect to provide ProQR with access to the company’s proprietary delivery technology for its wholly owned pipeline.

Based on its original September 2021 agreement and the expanded agreement announced today with Lilly, in total, ProQR is eligible to receive up to approximately $3.75 billion in research, development and commercialization milestones, as well as tiered royalties of up to mid-single digit percentage on product sales.

About Axiomer

ProQR is pioneering a next-generation RNA base editing technology called Axiomer, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer "Editing Oligonucleotides", or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.

On December 22, 2022 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of conditionally activated oncology therapeutics, reported that it plans to outline its pipeline priorities for 2023 and provide a data update for the CX-2029 Phase 2 cohort expansion study, on Thursday, January 5, 2023, after the close of U.S. markets (Press release, CytomX Therapeutics, DEC 22, 2022, View Source [SID1234625533]). Following the announcement, the Company will host a conference call and webcast at 5:00 p.m. ET / 2:00 p.m. PT to discuss updates.

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Participants may access the live webcast of the conference call from the Events and Presentations page of CytomX’s website at View Source Participants may register for the conference call here and are advised to do so at least 10 minutes prior to joining the call. An archived replay of the webcast will be available on the Company’s website.

On December 22, 2022 Chimeric Therapeutics, a clinical stage cell therapy company and an Australian leader in cell therapy, reported the successful completion of the planned dosing of the third patient cohort (n=3) in the Phase 1 dose escalation study evaluating the safety and maximum tolerated dose of Chimeric’s CHM 1101 (CLTX CAR T) cell therapy, in patients with recurrent or progressive glioblastoma (GBM) (Press release, Chimeric Therapeutics, DEC 22, 2022, View Source [SID1234625531]).

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The Phase 1A CHM 1101 clinical trial is taking place at City of Hope, one of the largest cancer research and treatment organizations in the United States. Chimeric Therapeutics has licensed the exclusive global rights to intellectual property covering the chlorotoxin CAR-T cells from City of Hope. Behnam Badie, M.D., City of Hope Chief of Division of Neurosurgery, is the trial’s principal investigator.

The Phase 1A study aims to enroll 18-36 patients with MMP2+ recurrent or progressive GBM across 4 dose levels. Study objectives are to evaluate the safety and efficacy of CLTX CAR T and to establish recommended dosing for a Phase 2 trial.

Patients (n=3) in this third dose level received a total dose of 240 X 106 CHM 1101 (CLTX CAR T) cells through dual routes of intratumoral and intraventricular administration.

Once the final evaluable patient of this third dose cohort successfully completes the 28 DLT period, the study will be able to advance to recruitment of patients at the fourth and final planned dose level of 440 X 106 CHM 1101 (CLTX CAR T) cells through dual routes of administration (intratumoral and intracranial intraventricular).

About CHM 1101 (Chlorotoxin CAR T):

CHM 1101, Chimeric’s Chlorotoxin CAR T (CLTX CAR T) is a first in class CAR T therapy that has the potential to address the high unmet medical need of patients with recurrent/ progressive glioblastoma.

CHM 1101 uniquely utilizes chlorotoxin (CLTX), a peptide derived from scorpion toxin, as the tumour-targeting component of the chimeric antigen receptor (CAR) which has been shown in preclinical models to bind more broadly and specifically to GBM cells than other targeting domains like EGFR, HER-2 or IL-13.

In preclinical models, CHM 1101 also demonstrated potent antitumor activity against glioblastoma while not exhibiting any off-tumor recognition of normal human cells/tissues, supporting a potentially optimal safety and efficacy profile.

On December 22, 2022 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that for the first time, a patient was dosed in the United States with its in-house manufactured product candidate UCART22, and completed the 28 day DLT period on December 14th, 2022, without complication (Press release, Cellectis, DEC 22, 2022, View Source [SID1234625530]).

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"First dosing of a patient with a product candidate manufactured in-house is a major milestone for Cellectis. UCART22 has been developed to potentially offer a therapeutic alternative for patients with r/r B-ALL, including patients that have relapsed from or unable to receive CD19-directed therapy. The ability to have our manufacturing completely in-house maximizes the chances that eligible patients can be treated without delay." said Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis.

"This is a transformational step forward for Cellectis: our in-house manufacturing capabilities would allow us to move product candidates like UCART22 from R&D to development to a finished UCART product on a timeline that would not have been possible working with a contract manufacturer," said Steven Doares, Senior Vice President, US Manufacturing & Raleigh Site Head. "We believe that having this capability in-house is a great competitive advantage as it would give us the ability to swiftly version our product candidates as we monitor clinical responses, resulting in what we expect to be the best product possible."

UCART22 is an allogeneic CAR T-cell product candidate that targets CD22 and is evaluated in the BALLI-01 clinical study, a Phase 1/2a open-label dose-escalation study designed to evaluate the safety and clinical activity of the product candidate in patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).

Three years ago, Cellectis made the decision to build its proprietary GMP manufacturing facilities in both Raleigh (North Carolina) and Paris to take control of its production and manufacturing timelines. Cellectis’ facilities are fully operational, showcasing the Company’s transformation into an end-to-end cell and gene therapy company, from discovery & product development, transfer, and cGMP manufacturing to clinical development.

As of now, Cellectis is one of the few end-to-end gene editing, allogeneic CAR T-cell companies that control its gene and cell therapy process from start to finish.

BALLI-01 is actively enrolling patients with relapsed or refractory B-ALL.

For more information, eligibility criteria and trial locations, please visit www.clinicaltrials.gov (NCT04150497) or contact [email protected].

About Acute Lymphoblastic Leukemia

Acute lymphoblastic leukemia (ALL) is a cancer of the lymphoid line of blood cells characterized by the development of large numbers of immature lymphocytes. ALL accounts for 0.3% of all new cancer cases, and 0.3% of all cancer deaths. It is estimated that 6,660 new cases of ALL and 1,560 deaths related to the disease occurred in the US in 2022. ALL represents 12% of all leukemia cases, progresses rapidly, and is typically fatal within weeks or months if left untreated.