On January 10, 2023 Defence Therapeutics Inc. ("Defence" or the "Company"), a Canadian biopharmaceutical company specialized in the development of immune-oncology vaccines and drug delivery technologies, is pleased to reported the advancement in the development of its AccumTM-mRNA vaccine program (Press release, Defence Therapeutics, JAN 10, 2023, View Source [SID1234626260]). This R&D program will not only impact the field of cancer immunotherapy, but it can also be directly applied to the development of new vaccines targeting infectious diseases.

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The mRNA vaccination approach offers tremendous advantages over the use of peptide-or protein-based vaccines. mRNA like any other biomolecule, is extremely sensitive to harsh conditions such as high acidity and enzymatic reactions, which would directly impede their therapeutic potency. In addition, mRNA molecules need to reach the cytoplasm where they can be efficiently translated into full proteins, this is where AccumTM may add stability and potency.

Defence is working with a private European company to synthesize mRNA vaccine coupled with its AccumTM. Defence’s has now completed the first phase of its AccumTM-mRNA vaccine development by achieving the synthesis and the Quality Control of the amino-modified polyA tail Ova mRNA. Defence is advancing on the second step, which consists of coupling AccumTM variants to amino-modified mRNA as well as testing and analyzing: i) the effect of AccumTM and linkers on mRNA stability, ii) the linker coupling onto amino-modified mRNA, iii) the AccumTM coupling onto linker-amino modified mRNA, and finally iv) the purification and analysis of the AccumTM-linker-amino-modified mRNA.

The third and final step of this Accum-mRNA vaccine development, scheduled at the end of January 2023, will be the production of a small vaccine batch to conduct in vivo studies in animals as a head-to-head comparison between AccumTM-linked and "naked" mRNA vaccines for their potential to generate an immune response capable of eradicating and controlling established tumors.

"The development of our AccumTM-mRNA vaccine is on track, our in vivo study on animals with pre-established solid tumors is planned for this quarter with the objective to demonstrate that AccumTM can significantly enhance the therapeutic potency of a given mRNA vaccine", says Mr. Plouffe, CEO of Defence Therapeutics.

The mRNA therapeutics market size is projected to surpass around USD 128.14 billion by 2030 and growing at a registered CAGR of 13.03% from 2022 to 2030 according to Precedence Research.

On January 10 ,2023 Bio4t2 reported that it dosed the first patient with T cells bearing a chimeric antigen receptor (CAR) that targets overexpression of BT-001 antigen present on various types of solid tumors (Press release, Bio4T2, JAN 10, 2023, View Source [SID1234626211]). The CAR-T (B4t2-001) was developed from Bio4t2’s PrismCore platform.

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"This first-in-human study marks the initial therapeutic to be evaluated from Bio4t2’s technology," said Dr. Laurence Cooper MD-PhD, Executive Chairman of the board. "PrismCore is capable of rapidly generating CAR-T to safely target self-antigens, opening a new frontier to delivering CAR-T to treat many types of solid tumors. This clinical trial is at the cutting edge of CAR-T biology and provides a path to treating the enormous numbers of patients who suffer from invasive cancers worldwide," added Dr. Cooper.

"We are excited to have started the clinical trial for our first CAR-T therapy targeting BT-001, which is a novel antigen for such therapies," said Farzad Haerizadeh, PhD, Chief Scientific Officer, and co-founder. "Our CAR-T is calibrated through PrismCore to discriminate between levels of BT-001 on tumors versus healthy cells. Indeed, B4t2-001 in preclinical studies in rodents and non-human primates, safely exhibited potent antitumor activity with long-term protective capacity. This trial helps validate the platform enabling the development of safe and effective CAR-T therapies against multiple types of solid tumors," said Haerizadeh.

About the clinical trial

The phase 1 investigator-initiated study (clinicaltrials.gov NCT05621486) evaluates ascending doses of B4t2-001 targeting BT-001 in patients with solid tumors. This trial assesses the safety, tolerability, pharmacokinetic, pharmacodynamic, and preliminary efficacy of autologous CAR-T as a single agent after lymphodepletion in adult subjects at Shanghai East and Shanghai Artemed hospitals in China.

On January 10, 2023 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported its 2023 financial guidance and commented on its outlook for the upcoming year (Press release, Halozyme, JAN 10, 2023, View Source [SID1234626179]).

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"In 2022, we project we achieved record revenue of greater than $655 million and we completed the acquisition of Antares Pharma, opening up an expanded set of opportunities for our subcutaneous drug delivery business," said Dr. Helen Torley, president and chief executive officer. "In 2023, we project record revenue of $815 to $845M and greater than 30% growth in EBITDA to $415 to $440 million. As our ENHANZE pipeline advances, we are excited about the future royalty revenue opportunities represented in 2023 by the potential for two new commercial launches, SC efgartigimod and SC atezolizumab."

Anticipated 2023 Key Events:

•Projected growth of >20% in the Company’s high-margin, recurring revenue stream from royalties to approximately $445 to $455 million driven by continued strength in Wave 2 products, including DARZALEX SC (daratumumab) and Phesgo (pertuzumab, trastuzumab and hyaluronidase) utilizing ENHANZE technology, as well as full year auto-injector royalty contribution.
•Two ENHANZE subcutaneous (SC) approvals and commercial launches by collaboration partners, including SC efgartigimod and SC atezolizumab.
•Four Phase 3 study data readouts from products utilizing ENHANZE by the Company’s collaboration partners
•SC efgartigimod in CIDP, ITP and Pemphigus (autoimmune disorders of the nervous system, blood and skin)
•SC ocrelizumab for certain patients with multiple sclerosis
•At least 2 new products utilizing ENHANZE entering Phase 1 development by the Company’s collaboration partners.
•At least one new collaboration and licensing agreement for ENHANZE, for a partner to test our developmental large volume auto-injector plus ENHANZE, and for our small volume auto-injector.
•Initiation of a clinical study to continue to evaluate the feasibility testing of our large volume auto-injector.

•Up to $150 million in share repurchases, dependent on market conditions and other factors, as part of the $750 million three-year share repurchase plan authorized by Halozyme’s board of directors in December 2021 demonstrating the Company’s continued commitment to a balanced capital allocation strategy.
2023 Financial Guidance:

•Revenue: Halozyme expects total revenue of $815 million to $845 million, representing growth of >20% over 2022 expected total revenue. The Company expects revenue from royalties to increase >20% over expected revenue from royalties in 2022, to a range of $445 million to $455 million.
•EBITDA: Halozyme expects EBITDA of $415 million to $440 million, representing growth of >30% over 2022 expected EBITDA and excludes the impact of amortization costs in 2023 related to the Antares Pharma acquisition.
•Earnings per Share: Halozyme expects Non-GAAP diluted earnings per share of $2.50 to $2.65. The Company’s earnings per share guidance does not consider the impact of potential future share repurchases.

Table 1. 2023 Financial Guidance
Guidance Range
Total Revenue $815 to $845 million
Royalty Revenue $445 to $455 million
EBITDA $415 to $440 million
Non-GAAP Diluted EPS $2.50 to $2.65

On January 10, 2023 Foundation Medicine, Inc., a pioneer in molecular profiling for cancer, reported a global collaboration with Karyopharm Therapeutics Inc. to develop FoundationOneCDx as a companion diagnostic for selinexor, which is being evaluated as a front-line maintenance therapy following systemic therapy in patients with advanced or recurrent TP53 wild-type endometrial cancer (Press release, Foundation Medicine, JAN 10, 2023, View Source [SID1234626171]).

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Endometrial cancer is the most common cancer of the female reproductive organs in the U.S.1 Roughly 50% of patients with advanced or recurrent disease have p53 wild-type tumors.2 Karyopharm’s phase 3 study (EC-042; NCT03555422) is a multicenter, blinded, placebo-controlled, randomized study evaluating the efficacy and safety of selinexor as a maintenance therapy following systemic therapy in patients with advanced or recurrent TP53 wild-type endometrial cancer. The study is utilizing Foundation Medicine’s tissue-based next generation sequencing test to identify and enroll patients whose tumors are TP53 wild-type.

"We’re looking forward to partnering with Karyopharm as they work to advance this exciting new treatment option for patients living with advanced endometrial cancer," said Sanket Agrawal, Chief Biopharma Business Officer, Foundation Medicine. "Our deep understanding of cancer biology and global regulatory expertise, combined with their innovative approach to developing cancer therapies puts us in a strong position to help more patients living with this devastating condition."

Foundation Medicine’s portfolio of FDA-approved comprehensive genomic profiling tests offers physicians both blood- and tissue-based testing options for detecting genomic alterations that help guide personalized treatment decisions. If approved, FoundationOne CDx would be the first companion diagnostic to identify patients with TP53 wild-type endometrial cancer who may be eligible for selinexor.

About Foundation Medicine: Your Essential Partner in Cancer Care

Foundation Medicine is a pioneer in molecular profiling for cancer, working to shape the future of clinical care and research. We collaborate with a broad range of partners across the cancer community and strive to set the standard for quality, scientific excellence, and regulatory leadership. Our deep understanding of cancer biology helps physicians make informed treatment decisions for their patients and empowers researchers to develop new medicines. Every day, we are driven to help our partners find answers and take action, enabling more people around the world to benefit from precision cancer care. For more information, please visit us on www.FoundationMedicine.com and follow us on Twitter and LinkedIn.

On January 10, 2023 -NextPoint Therapeutics, a biotechnology company developing a new world of precision immuno-oncology, reported that it raised $80 million in Series B financing co-led by Leaps by Bayer, the impact investment arm of Bayer AG, and Sanofi Ventures, the strategic venture capital arm for Sanofi (Press release, NextPoint Therapeutics, JAN 10, 2023, View Source [SID1234626170]). The financing will be used to advance NextPoint’s two lead precision immuno-oncology programs into the clinic, both targeting the newly discovered HHLA2 pathway to activate anti-tumor immune responses.

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Additional new investors in the round include Invus, Catalio Capital Management, Sixty Degree Capital and PagodaTree Partners. Existing investors that took part in the financing include MPM Capital Management, Binney Street Capital/Dana-Farber Cancer Institute and NextPoint founder Gordon Freeman, PhD. As part of the financing, Rakhshita Dhar, Senior Director of Venture Investments Health at Leaps by Bayer, and Paulina Hill, Partner at Sanofi Ventures, will join the NextPoint Board of Directors.

NextPoint’s programs aim to deliver monotherapies for cancer patients without viable treatment options. While immune checkpoint inhibitors targeting PD-1/L1 have revolutionized cancer treatments, many patients do not benefit from these medications and require novel therapeutic strategies. Similar to PD-L1, the tumor antigen HHLA2 is a member of the B7 receptor family, is highly expressed on certain hard-to-treat cancers, and drives avoidance of detection from the immune system. Importantly, HHLA2 is independent of PD-L1 and is often most strongly expressed in PD-L1-negative cancers. NextPoint’s approach re-activates immune cells in tumors that are suppressed by HHLA2-driven immune evasion.

NextPoint originated from the combined expertise of its academic founders, Gordon Freeman, PhD, of the Dana-Farber Cancer Institute, and XingXing Zang, PhD, of Albert Einstein College of Medicine. Drs. Freeman’s and Zang’s independent discovery and characterization of the HHLA2 pathway formed the basis of the NextPoint approach. The company and its founders have shown in preclinical models and with analysis of existing clinical datasets that the HHLA2 pathway is an important tumor-suppressive mechanism in many patients1,2.

"NextPoint is building a deep understanding of the HHLA2 tumor-specific immune-escape mechanism, with the ultimate goal of establishing standalone treatments in cancers with high HHLA2 expression," said Detlev Biniszkiewicz, PhD, Chief Executive Officer of NextPoint Therapeutics. "The support of our new investors along with the continued commitment of our existing investors and founders emphasizes our momentum and progress in defining precision immuno-oncology for new patient segments."

Juergen Eckhardt, MD, Head of Leaps by Bayer, commented, "Leaps by Bayer was founded to help solve ten of the world’s biggest challenges in health and agriculture, including preventing and curing cancer. We are thrilled to support NextPoint, an exciting addition to our oncology portfolio, as it works to redefine the treatment landscape of immuno-oncology."