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.

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.

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.

On September 28, 2022 Pheon Therapeutics (Pheon), a leading Antibody-Drug Conjugate (ADC) specialist developing next generation ADCs for a wide range of hard-to-treat cancers, reported that launched following the closing of a $68 million Series A financing in March 2022 (Press release, Pheon Therapeutics, SEP 28, 2022, View Source [SID1234621482]). The investment will enable Pheon to advance its lead ADC program to clinical proof-of-concept and establish a pipeline of novel ADCs. The financing was led by Brandon Capital, Forbion and Atlas Venture, with participation from seed investor Research Corporation Technologies (RCT).

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Pheon’s lead program exploits a novel target that is highly expressed in a broad range of solid tumors. The lead compound is expected to reach IND within the next 18 months. Understanding there is no "one size fits all" in engineering ADCs, Pheon takes a methodical approach to ADC development. Using both novel and clinically validated mAbs and arming them either with warheads from its proprietary payload platform which boasts a novel mechanism of action or with off-the-shelf linker payload combinations, Pheon is finely attuned to balancing safety and efficacy for each target.

Chief Executive Officer Bertrand Damour heads the Company’s leadership team which includes industry veteran Leigh Zawel as Chief Scientific Officer. Pheon’s co-founders include Paul Jackson, Vice President R&D, and advisor Professor David Thurston. Professor Thurston was previously co-founder of Spirogen, whose payload technology is contained within ZynlontaTM, a recently approved ADC.

Bertrand Damour has over 20 years’ management experience in both the European and US biotech industries having been the CEO of several companies. He was previously CEO of NBE Therapeutics, the developer of NBE-002, an anti-ROR1 ADC which Boehringer Ingelheim acquired for $1.4 billion in 2020. Earlier in his career, he was CEO of GeneProt Inc., a US proteomics company; Mind NRG, a Swiss biotech in the field of CNS (which was acquired by Minerva Neurosciences); OncoEthix, a Swiss oncology company in the field of epigenetics, which was acquired by Merck & Co. for $375 million; and Synthena AG, a company developing oligonucleotide-based therapeutics for the treatment of genetic neuromuscular diseases. Bertrand has also held senior roles within the banking industry and has extensive expertise in corporate transactions including M&A, LBOs and IPOs, which he gained while working at JP Morgan, Rabobank International and Deutsche Bank in New York.

Bertrand Damour, Chief Executive Officer of Pheon Therapeutics, said: "Pheon is developing a first-in-class ADC and has built a highly differentiated proprietary payload platform which is gaining strong momentum. We are laser-focused on implementing our strategy to get our first program into clinical development as rapidly as possible and the preclinical data generated so far are very promising. The track record and expertise of the leadership team at Pheon is outstanding and I am proud to be working with them on this innovative approach to developing treatments for cancer patients."

Dr Leigh Zawel has over two decades of oncology drug discovery experience and was previously the CSO at NASDAQ-listed Cullinan Oncology where he oversaw the development of CLN-081, a best-in-class Exon20 EGFR TKI that was ultimately partnered with Taiho Pharmaceuticals and Zai Labs. Prior to Cullinan, Dr Zawel was Vice President and East Coast Site Head for Pfizer’s Centers for Therapeutic Innovation, a group that achieved four INDs in the four years under his remit. As an Oncology Site Head at Merck in the early 2010s he led the teams that brought the first ERK and P53/MDM2 inhibitors into the clinic. Dr Zawel also worked at the Novartis Institutes for Biomedical Research, where he brought one of the first IAP inhibitor compounds into early clinical development. Dr Zawel earned his PhD in the laboratory led by Professor of Biochemistry and Molecular Pharmacology at the New York University School of Medicine, Danny Reinberg, a biochemist whose contributions have impacted the fields of mammalian transcription, gene expression and epigenetics. Dr Zawel has published more than 35 scientific publications and is an author on 45 patents.

Dr Leigh Zawel, Chief Scientific Officer of Pheon Therapeutics, said: "ADC technology has reached an inflection point. At Pheon, I’m thrilled to be working with Bertrand and with the team that we have assembled which has deep technical expertise and the resources to develop the next generation of ADCs to positively impact the lives of patients with cancer."

On behalf of the Board, Chair Jonathan Tobin and Partner at Brandon Capital, said: "In the last few years ADC drugs have started to show unprecedented clinical efficacy through a better understanding of the properties that make an effective ADC, combined with next generation payloads and targets. We are excited by the potential of Pheon’s first-in-class antibody target coupled with its novel proprietary payload platform to make a significant contribution to the ADC field and cancer patients. Pheon has attracted a strong syndicate of investors with the likes of Brandon Capital, Forbion and Atlas, and a proven leadership team focused on rapidly bringing its first-in-class ADC to the clinic. We look forward to extending the benefit of these cutting-edge therapeutics to more patients through target and payload innovation at Pheon."

The Company’s Board includes Michael Gladstone, Partner at Atlas Venture; Jonathan Tobin, Chair, Partner at Brandon Capital; Rogier Rooswinkel, General Partner at Forbion; and Shaun Kirkpatrick, President Biotechnologies at Research Corporation Technologies (RCT) and Bertrand Damour, CEO of Pheon Therapeutics.

On September 27, 2022 Avid Bioservices, Inc. (NASDAQ:CDMO), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that the company will participate in the RBC Global CDMO Conference. Nick Green, president and chief executive officer, will be the featured speaker in a fireside chat at the conference, which will take place October 3-4, 2022 (Press release, Avid Bioservices, SEP 27, 2022, View Source [SID1234621515]).

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Details of the company’s participation are as follows:

RBC Global CDMO Conference

To listen to the webcast of the RBC fireside chat please visit: View Source