Aura Biosciences Announces First Patient Dosed in Phase 1 Study Evaluating Belzupacap Sarotalocan (AU-011) for the Treatment of Non-Muscle Invasive Bladder Cancer

On September 28, 2022 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported the first patient has been dosed in a Phase 1 study evaluating belzupacap sarotalocan, the Company’s first VDC product candidate, for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC) (Press release, Aura Biosciences, SEP 28, 2022, View Source [SID1234621494]).

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"Dosing of the first patient in this Phase 1 study is an exciting key milestone both for Aura and for the field of urologic oncology, as approximately 70% of patients with bladder cancer globally are diagnosed early with NMIBC," said Dr. Cadmus Rich, Chief Medical Officer and Head of R&D of Aura Biosciences. "There have been no major advances in the early treatment of NMIBC in over two decades. We look forward to presenting initial Phase 1 data in 2023 and advancing the development of a potential new therapeutic option for patients with a high unmet medical need."

The Phase 1 multi-center, open label clinical trial is expected to enroll approximately 23 adult patients with NMIBC. The trial is designed to assess the safety and tolerability of belzupacap sarotalocan as a single agent. The primary endpoint of the Phase 1 clinical trial is the incidence and severity of treatment-related adverse events and serious adverse events and the incidence of dose-limiting toxicities. Aura anticipates presenting initial Phase 1 data in 2023. The U.S. Food and Drug Administration (FDA) granted Fast Track designation for belzupacap sarotalocan in June 2022. The opportunity for more frequent interactions with Division of Oncology at the FDA and the potential for Priority Review will be valuable as belzupacap sarotalocan advances further into clinical development in patients with NMIBC.

Press Release: Availability of the Q3 2022 Memorandum for modelling purposes

On September 28, 2022 Sanofi reported that its Q3 2022 Memorandum for modelling purposes is available on the "Investors" page of the company’s website (Press release, Sanofi, SEP 28, 2022, View Source [SID1234621493]):

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As for each quarter, Sanofi prepared this document to assist in the financial modelling of the Group’s quarterly results. This document includes a reminder on various non-comparable items and exclusivity losses as well as the foreign currency impact and share count. Sanofi’s third-quarter 2022 results will be published on October 28, 2022.

Philogen’s management team will hold a webinar on 29 September 2022 at 16:00 CEST

On September 28, 2022 Philogen reported that it’s management team will hold a webinar on 29 September 2022 at following an R&D Update (Press release, Philogen, SEP 28, 2022, View Source [SID1234621492]).

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Prof. Dr. Dario Neri, Chief Executive Officer, Dr. Laura Baldi, Chief Financial Officer, and Dr. Emanuele Puca, Head of Investor Relations, will host a 1-hour live webcast and conference call to provide an update on Philogen clinical programs. The presentation will be followed by a Q&A session.

CRISPR Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX130™ for the Treatment of Cutaneous T-Cell Lymphomas (CTCL)

On September 28, 2022 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX130, the Company’s wholly-owned allogeneic CAR T cell therapy targeting CD70, for the treatment of Mycosis Fungoides and Sézary Syndrome (MF/SS) (Press release, CRISPR Therapeutics, SEP 28, 2022, View Source [SID1234621491]).

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"The RMAT designation is an important milestone for the CTX130 program that recognizes the transformative potential of our cell therapy in patients with T-cell lymphomas based upon encouraging clinical data to date," said Phuong Khanh (P.K.) Morrow, M.D., FACP, Chief Medical Officer of CRISPR Therapeutics. "We continue to work with a sense of urgency to bring our broad portfolio of allogeneic cell therapies to patients in need."

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

About CTX130 and COBALT Trials
CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting Cluster of Differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1 single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 in adult patients. The COBALT-LYM trial is evaluating the safety and efficacy of CTX130 for the treatment of relapsed or refractory T or B cell malignancies. The COBALT-RCC trial is evaluating the safety and efficacy of CTX130 for the treatment of relapsed or refractory renal cell carcinoma. CTX130 has received Orphan Drug and Regenerative Medicine Advanced Therapy designations from the FDA.

Race Develops Improved IV Formulation of Zantrene

On September 28, 2022 Race Oncology Limited ("Race") reported that Race researchers, led by Dr Benjamin Buckley in collaboration with the University of Wollongong (ASX Announcement: 8 November 2021), have developed a new formulation of Zantrene that enables peripheral (arm or leg vein) intravenous (IV) delivery to patients (Press release, Race Oncology, SEP 28, 2022, View Source [SID1234621489]). This novel and improved formulation (codename RC220) provides clinicians with an easier to use alternative to the current central line formulation of Zantrene. This formulation has greater market potential and is particularly well suited to solid tumour patients.

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Clinical Significance
Administration of Zantrene has until now required the use of an invasive central venous catheter (central or main line) that must be performed in a hospital setting. While this is common practice for the delivery of chemotherapy drugs in patients with leukaemias, it is not optimal for patients with solid tumours (such as breast cancer, melanoma, lung cancer, kidney cancer, etc) where peripheral IV infusion in an outpatient setting is often preferred by both the patient and treating oncologist.

Peripheral IV administration can provide a better quality of life for patients with less pain and lifestyle disruption as it enables patients to be treated outside of a major hospital or within their own homes. The ability to precisely match the required drug dose to the patient’s need is also simpler using an IV formulation than with other delivery options, such as fixed size oral dosing. In addition, for uses where low and continuous dosing are desirable (such as inhibiting an enzyme like FTO), a peripheral IV formulation can be a better option.

Commercial Significance

Peripheral IV formulations have significant commercial advantage. As central line administration requires highly skilled healthcare personnel, simpler peripheral IV formulations are attractive in resource constrained healthcare environments. An additional commercial benefit of the new peripheral IV formulation is the ability to deliver Zantrene more rapidly to the patient, minimising occupancy of expensive oncology infusion chairs and providing the patient with a better treatment experience. Race Oncology Ltd ABN 61 149 318 749 Registered office: L36, 1 Macquarie Place, Sydney NSW 2000 www.raceoncology.com A more immediate impact of the RC220 IV formulation is improved patient recruitment potential for future clinical trials. Only a minority of solid tumour patients are willing to participate in clinical trials that require central line infusions.

In a competitive oncology trial environment where the patient has many suitable trials to choose between, comfort and quality of life are important non-clinical factors that strongly influence patient choice of trial enrolment. Next Steps Race has signed a contract with Societal (San Diego, California, USA) to produce the new RC220 IV formulation to the FDA current Good Manufacturing Practice (cGMP) standard that is required for use in clinical trials. Societal are already familiar with Zantrene as they are the manufacturer of the current central line formulation. The final RC220 drug product is expected to be delivered by late Q2 2023 at a contracted cost of US$611,900.

A new international patent will be submitted in early Q2 2023. Advice from Race’s patent attorneys is that the RC220 formulation is both novel and inventive and it should secure international patent protection. Delaying patent submission for as long as possible maximises the effective on-patent life of RC220 and hence its future commercial value. Collaborative activities are continuing at the University of Wollongong and other sites to develop additional formulations of Zantrene that can be delivered orally and/or less frequently (i.e. longer acting formulations).

Race will update investors on the progress of these new formulation activities when completed. Race CSO Dr Daniel Tillett said: "The development of the RC220 IV formulation is a major advance for Race. The chemical properties of Zantrene make developing a peripheral formulation highly complex and challenging. I am extremely proud of the Race team and our collaborators in developing the new RC220 formulation and believe this is a pivotal step in bringing the promise of Zantrene to clinical and commercial reality."

Race CMO Dr David Fuller said: "The availability of a peripherally administered formulation of Zantrene is a major step forward and will allow Race to more effectively explore multiple opportunities in the solid tumour and cardioprotection space." Race Oncology Ltd ABN 61 149 318 749 Registered office: L36, 1 Macquarie Place, Sydney NSW 2000 www.raceoncology.com Q & A What clinical problem does the new RC220 IV Zantrene formulation solve? Zantrene is very insoluble in the blood. It was discovered early in clinical development that if Zantrene is infused via a peripheral vein it will crystallise and block the flow of blood through the vein.

This problem was solved by using a central line catheter to slowly infuse into a large blood vessel near the heart (aorta) over 2 hours, where the high flow of blood diluted the Zantrene before it had a chance to crystallise. While this approach works, it is not ideal for many patients and clinicians. The new RC220 formulation is able to keep Zantrene from crystallising in the blood even when it is infused into a smaller peripheral arm or leg vein.

The RC220 formulation also allows Zantrene to be infused faster improving the patient’s treatment experience. What does this formulation breakthrough mean for Race shareholders? The RC220 formulation is important for three reasons: 1. It expands the potential market size for Zantrene by making it easier to use in clinical practice, especially with non-leukaemia cancers. The value of any new drug is a function of the market size, the share that can be captured, and the drug price. 2. It makes it easier to recruit patients into our clinical trials, which will reduce the cost and time to complete these trials. 3. It provides valuable and protectable IP that effectively resets the patent clock on Zantrene. This ‘improved formulation’ approach is widely used in the pharmaceutical industry to protect drugs that have reached the end of life of their original patents. A good example of this in oncology was the development of Abraxane, a protein bound formulation of paclitaxel (Taxol). This formulation avoided some of the side effects caused by the original Cremophor EL-based formulation. The developers of Abraxane, Abraxis BioScience, were acquired by Celgene in 2009 for US$2.9 billion, 10 times the annual sales of Abraxane at the time1. An old drug with an improved formulation can be highly valuable.