On March 9, 2023 REVEAL GENOMICS, S.L., a Barcelona-based biotechnology start-up seeking to revolutionize precision oncology through biomarker innovation, reported a new addition to its pipeline consisting of a novel biomarker approach in liquid biopsy for patients with advanced cancer (Press release, REVEAL GENOMICS, MAR 9, 2023, View Source [SID1234628475]). The main findings of this development were published in Nature Communications this month, in collaboration with several academic institutions, including the Hospital Clinic/IDIBAPS de Barcelona (Spain), University of Barcelona (Spain), Vall d’Hebrón Institute of Oncology (Barcelona, Spain), Hospital Universitario 12 de Octubre (Madrid, Spain), the Catalan Institute of Oncology (Badalona, Spain), and the University of North Carolina (Chapel Hill, USA).

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Analysis of cell-free DNA (cfDNA), present in body fluids such as plasma, is broadly known as liquid biopsy. In oncological patients, cfDNA contains a fraction of tumor-derived genomic material (ctDNA), that has been proven as a valuable resource to identify tumor-borne genetic alterations. However, cancer is highly complex, and additional biological information is needed to refine the prediction of patient outcome and/or benefit from treatment.

Dr. Aleix Prat, Co-Founder and CSO of REVEAL GENOMICS and the lead author of the study, explained: "Our proprietary and novel supervised learning computational approach predicts complex tumor features including gene expression, protein and tumor histology using one data source: DNA sequencing. Here, we demonstrate that our approach is feasible in plasma cfDNA and that it provides clinically relevant information."

New product in the pipeline
Single DNA alterations in metastatic breast cancer, such as ESR1 or PIK3CA mutations, are valuable information but are not enough to predict the patient outcome. To address this issue, investigators from REVEAL GENOMICS sequenced cfDNA from 459 patients with metastatic breast cancer, including 245 patients with hormone receptor-positive/HER2-negative (HR+/HER2-) treated with endocrine therapy and a CDK4/6 inhibitor (i.e., palbociclib, ribociclib or abemaciclib).

Firstly, REVEAL GENOMICS’ proprietary machine-learning multi-gene signatures were applied in plasma and linked to DNA/RNA/protein data from tumor tissue (paired samples). The results showed, for the first time, that multi-gene signatures tracking complex biological features can be successfully identified in ctDNA. These complex biological features include, among others, measures of tumor proliferation and estrogen receptor signaling, similar to what is accomplished using direct tumor tissue DNA or RNA profiling. For example, one of REVEAL GENOMICS´ signatures in plasma ctDNA, known as the estrogen receptor (ER) signaling signature, predicted the ER status of the tumor tissue as determined by standard immunohistochemistry.

Secondly, the authors discovered that the combination of 150 biologically relevant signatures identified 4 new DNA-based molecular subtypes of breast cancer. More importantly, these 4 biological entities were strongly associated with survival outcome in patients with early-stage and/or metastatic HR+/HER2- breast cancer.

Finally, the investigators focused on a particular genomic signature called retinoblastoma loss-of-heterozygosity (RB-LOH), which was designed to capture tumor biological status induced by the loss of RB – a known key player in tumor cell division needed for CDK4/6 inhibitors to work. In patients with metastatic HR+/HER2- breast cancer treated with endocrine therapy and a CDK4/6 inhibitor, REVEAL GENOMICS’ ctDNA RB-LOH signature identified the 20-30% of patients who do not respond well to this treatment.

Dr. Ana Vivancos, Co-Founder and consultant of REVEAL GENOMICS, concluded: "Our novel approach can have huge implications in the clinical setting, and opens new opportunities for the discovery of multi-feature genomic predictors coming from ctDNA in breast cancer and other types of cancer."

About liquid biopsy
The liquid biopsy market in oncology can potentially become the future gold standard, considering its various advantages over conventional cancer diagnostic approaches. The global market boasts a total value of $4.3 billion in 2022 and a projected growth rate of 18.3%, reaching $10.0 billion by 2027. The market is driven by the rising incidence and prevalence of cancer in the developing world and the increasing preference for noninvasive treatment procedures.

Dr. Patricia Villagrasa, Co-Founder and CEO of REVEAL GENOMICS, concluded: "Our company is committed to creating and developing novel genomic-based assays in oncology and demonstrating their clinical utility as we have already done with our first product, the HER2DX, which is already impacting patient care in early-stage HER2-positive breast cancer. This new ctDNA-based assay, called DNADX, is our second product expected to be available in 2024." The Spanish Ministry of Science and Innovation has granted funding for this new product, validating DNADX as an innovative and clinically useful tool.

On March 9, 2023 Allorion Therapeutics ("Allorion"), a biotech company focusing on next-generation precision medicine for oncology and autoimmune diseases, reported the completion of a $50 million Series B financing round, co-led by INCE Capital and Qiming Venture Partners, with participation from TF Capital, Long River Investment (LRI), 3SBio, Octagon Capital, and Elikon Venture (Press release, Allorion Therapeutics, MAR 9, 2023, View Source [SID1234628474]). Since its establishment in July 2020, Allorion has raised over $100 million from reputable institutional biotech investors.

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The funds raised in this round will primarily support upcoming Phase I/II clinical trials in both China and the U.S. and the identification of clinical candidates for additional programs. Allorion will also continue to advance its established and validated early discovery platform technologies, including an allosteric inhibitor screening platform and a platform for discovery of synthetic lethality targets and molecules. The funds will also enable Allorion to further build world leading clinical development and business development teams.

As the Series B financing round concludes, Dr. Gregory Berk has officially joined Allorion as the Chief Medical Officer (CMO). Dr. Berk is a clinical research expert with over 30 years of experience in novel drug development. He will be responsible for building clinical teams in the U.S. and China, as well as designing and overseeing the Phase I/II clinical trials in both countries. Before joining Allorion, Dr. Berk held senior management positions as SVP or CMO at several renowned biotech/biopharma companies in the U.S., including Abraxis BioScience, Intellikine, BIND BioSciences, Sideris Pharmaceuticals, and GT Biopharma. His extensive clinical trial experience and industry network will help Allorion transition from preclinical research to the clinical development stage, developing new drugs to address significant unmet medical needs while creating value for the company.

Regarding the successful Series B financing, Peter Ding, Allorion’s co-founder and CEO commented, "We sincerely appreciate the recognition of and support from both new and existing investors who participated in this round of financing. Since its launch in July 2020, Allorion has established wet labs in both Boston and Guangzhou, and currently has a strong R&D team of more than 50 full-time employees. In just two and a half years, the company has advanced two small molecules to IND stage in both China and the U.S. Our highly innovative technology platforms have generated first-in-class small molecule programs with immense clinical and commercial value, some of which have progressed to the clinical candidate selection stage. Allorion is also actively pursuing collaboration and co-development opportunities with several global pharma companies, who have shown great interest in our programs at this year’s JPM conference, demonstrating a global recognition of our innovation. Upholding a science- and data-driven R&D strategy, we will continue to fully leverage our team’s capabilities, platforms, and funds to advance clinical development and address unmet medical needs for patients."

JP Gan, the founding partner of INCE Capital, said, "With outstanding industry experience and excellent execution, Allorion has built and validated several world-leading early discovery platforms for next-generation precision medicines, including an allosteric inhibitor screening platform, a synthetic lethality target and molecule discovery platform, and an AI-enabled drug design platform. The team has also successfully advanced two molecules discovered by these platforms to the IND stage. We look forward to the clinical data of its leading assets and believe that the company’s platforms will continue to generate highly innovative pre-clinical candidates to address significant unmet medical needs and serve patients around the world."

Kan Chen, Partner at Qiming Venture Partners commented, "Allorion team consists of industry veterans from well-known biopharmaceutical companies such as Novartis, Merck, and Eli Lilly, who boast rich experience in small molecule drug discovery. The team has advanced two molecules to the clinical stage and verified the company’s allosteric inhibitor and synthetic lethality screening platforms, demonstrating its strong execution capability. We are very much looking forward to the clinical data, additional high-quality drug candidates generated by the company’s technology platforms, and its collaboration with international and Chinese pharma, driving more innovations valuable to human health."

Wei Cheng, Managing Director of TF Capital, commented, "It’s very impressive that Allorion built a strong team with global competitiveness in just two and a half years. TF Capital is proud to support Allorion from angel round all the way through the B financing round and is happy to witness the company deliver exciting results one after another. Successfully entering the clinical stage is a critical milestone. We look forward to Allorion’s continued progress in early drug discovery, clinical studies, and business collaboration, ultimately benefiting patients all around the world."

On March 9, 2023 Onconova Therapeutics, Inc. (NASDAQ: ONTX), ("Onconova"), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported that the Company intends to release its fourth quarter and full year 2022 financial results on Thursday, March 16, 2023 (Press release, Onconova Therapeutics, MAR 9, 2023, View Source [SID1234628472]). Management plans to host a conference call and live webcast at 4:30 p.m. ET on the same day to discuss these results and provide an update on its pipeline programs.

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Conference Call and Webcast Information

Interested parties who wish to participate in the conference call may do so by dialing (800) 715-9871 for domestic and (646) 307-1963 for international callers and using conference ID 3097517.

Those interested in listening to the conference call via the internet may do so by visiting the investors and media page on the Company’s website at www.onconova.com and clicking on the webcast link. In addition to the live webcast, a replay will be available on the Onconova website for 90 days following the call.

On March 9, 2023 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, reported updates and financial results for the fourth quarter and full year ended December 31, 2022 (Press release, Poseida Therapeutics, MAR 9, 2023, View Source [SID1234628471]).

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"For Poseida, 2022 was a year of execution, progress and validation for our proprietary technology and programs in both cell and gene therapy. In cell therapy, we advanced two fully allogeneic CAR-T programs into the clinic and presented early clinical data for both programs at ESMO (Free ESMO Whitepaper) I-O in December 2022. We also established a strategic partnership with Roche, a global leader in oncology, focused on our allogeneic CAR-T platform in hematological indications," said Mark Gergen, Chief Executive Officer of the Company. "In gene therapy, our Takeda-partnered in vivo gene therapy program in Hemophilia A continued to demonstrate promise as a potential functional cure with proof of principle data presented at ASH (Free ASH Whitepaper). Along with the rest of the industry, we continue to manage through headwinds as we deal with macro-economic issues and conditions that can impact multiple aspects of our business. We remain focused on advancing our business and continuing to drive innovation as reflected at our recent R&D Day."

Program Updates

CAR-T Programs
In cell therapy, the Company is focused on three allogeneic CAR-T programs with two programs currently progressing in Phase 1 clinical trials and one expected IND during the year:

MUC1-C Program
P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate targeting solid tumors derived from epithelial cells, including breast and ovarian cancers. The Company is currently evaluating P-MUC1C-ALLO1 in a Phase 1 clinical trial and presented early clinical data in December 2022 at ESMO (Free ESMO Whitepaper) I-O in Geneva, Switzerland. Data highlighted at the meeting showed that P-MUC1C-ALLO1 was well tolerated, with no dose-limiting toxicities (DLTs), cytokine release syndrome (CRS), graft vs host disease (GVHD) or immune effector cell-associated neurotoxicity syndrome (ICANS). In addition, P-MUC1C-ALLO1 showed encouraging clinical activity, including an objective partial response in a breast cancer patient at the lowest dose. The Company expects to present further clinical data updates for the program at a medical meeting in 2023.

BCMA Program
P-BCMA-ALLO1 is an allogeneic CAR-T product candidate being developed to target relapsed/refractory multiple myeloma (R/R MM) in partnership with Roche. The Company is currently evaluating P-BCMA-ALLO1 in a Phase 1 clinical trial and shared early clinical data from the program at ESMO (Free ESMO Whitepaper) I-O in December 2022. P-BCMA-ALLO1 was well tolerated, with no DLTs, CRS, GVHD or ICANS. P-BCMA-ALLO1 demonstrated responses in heavily pre-treated patients with R/R MM at the lowest dose, including in patients who had previously received BCMA-targeted therapy and patients with high-risk disease. In the fourth quarter of 2022, the Company also successfully added the ability to manufacture P-BCMA-ALLO1 at its internal GMP pilot plant in San Diego. The Company expects to present further clinical updates for the program at a medical meeting in 2023, subject to clearance with Roche.

CD19CD20 Program
P-CD19CD20-ALL01 is a preclinical allogeneic CAR-T product being developed to target B-cell malignancies in partnership with Roche. P-CD19CD20-ALL01 is the Company’s first dual CAR program and contains two fully functional CAR molecules to target cells that express either CD19 or CD20. The Company believes that by targeting both CD19 and CD20, there is potential to overcome some of the issues of earlier generation CD19 CAR-T products where antigen escape has been observed. The Company expects to file an IND for P-CD19CD20-ALLO1 in mid-2023.

Gene Therapy Programs
The Company is advancing multiple preclinical gene therapy programs in liver-directed diseases:

OTC Program
P-OTC-101 is an in vivo program for the treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene. The Company is developing the P-OTC-101 program utilizing a hybrid delivery system and working on an updated timeline for the program. The Company presented data at its R&D Day in February 2023, highlighting continued advancements in preclinical models leading towards a potential functional cure of OTC Deficiency.

FVIII Program
The Company is advancing its P-FVIII-101 preclinical program partnered with Takeda, which is in development for the in vivo treatment of Hemophilia A. P-FVIII-101 utilizes piggyBac gene modification delivered via lipid nanoparticle that has demonstrated stable and sustained Factor VIII expression in animal models. The Company presented preclinical data from this program at the ASH (Free ASH Whitepaper) annual meeting in New Orleans in December 2022, which showed that P-FVIII-101 achieved and sustained normalized (>50%) human Factor VIII activity following a single dose and delivered therapeutic Factor VIII activity in mice following single and repeat doses, indicating the potential for dose titration. The data support that, with the Company’s piggyBac delivery system, the therapeutic transgene expression cassette can be stably integrated into the genome of liver cells and provide consistent and durable therapeutic activity.

PAH Program
Announced at the Company’s R&D Day in February 2023, P-PAH-101 is a liver-directed gene therapy to treat Phenylketonuria (PKU), an inherited genetic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene resulting in buildup of phenylalanine in the body. If left untreated, PKU can affect a person’s cognitive development. P-PAH-101 utilizes piggyBac technology combined with its hybrid adeno-associated virus (AAV) and nanoparticle delivery system. The Company’s preclinical data has demonstrated the potential to resolve phenylalanine to normal levels following a single treatment in juvenile and adult mice. P-PAH-101 is partnered with Takeda and currently in preclinical development.

Expansion of Gene Therapy Scientific Advisory Board
In 2022 the Company announced the formation of its Gene Therapy Scientific Advisory Board (SAB), chaired by George M. Church, Ph.D. In March 2023, the Company announced the appointment of five new members to its Gene Therapy SAB to provide counsel on the Company’s gene therapy programs in development.

Financial Results for the Fourth Quarter and Full Year 2022
Revenues
Revenues were $10.1 million and $130.5 million for the fourth quarter and the full year ended December 31, 2022, compared to $31.2 million for both the fourth quarter and the full year ended December 31, 2021. The increase of revenues was primarily due to revenues earned from the collaboration and license agreement with Roche, which became effective in the third quarter of 2022, and the Takeda collaboration and license agreement, which became effective in the fourth quarter of 2021.

Research and Development Expenses
Research and development expenses were $33.9 million for the fourth quarter ended December 31, 2022, compared to $39.1 million for the same period in 2021. The decrease was primarily driven by the wind-down of the Company’s P-BCMA-101 and P-PSMA-101 autologous programs as the Company transitioned to an allogeneic platform, offset by an increase in personnel expenses as a result of increased headcount, which included an increase in stock-based compensation expense.

For the full year ended December 31, 2022, research and development expenses were $152.9 million, compared to $136.7 million for the same period in 2021. The increase was primarily due to an increase in personnel expenses as a result of increased headcount, which included an increase in stock-based compensation expense, and an increase in overall active clinical programs offset by the wind-down of the Company’s P-BCMA-101 autologous program as the Company transitioned to the allogeneic program.

General and Administrative Expenses
General and administrative expenses for the fourth quarter ended December 31, 2022 and 2021, were $9.4 million and $9.6 million, respectively. The decrease was primarily related to a decrease in insurance expense, offset by an increase in personnel expenses due to an increase in headcount.

For the full year ended December 31, 2022 and 2021, general and administrative expenses were $37.5 million and $35.9 million, respectively. The increase was primarily related to an increase in personnel expenses due to an increase in headcount, which included an increase in stock-based compensation expense.

Net Income (Loss)
Net loss was $33.3 million for the fourth quarter ended December 31, 2022, compared to a net income of $1.5 million for the same period of 2021. Net loss for the full year ended December 31, 2022 and 2021, were $64.0 million and $125.0 million, respectively.

Cash Position
As of December 31, 2022, the Company’s cash, cash equivalents and short-term investments balance was $282.5 million. The Company expects that its cash, cash equivalents and short-term investments together with the remaining near-term milestones and other payments from Roche will be sufficient to fund operations into at least mid-2024.

On March 9, 2023 MorphoSys AG (FSE: MOR; NASDAQ: MOR) reported thgat it will publish its results for the financial year 2022 on March 15, 2023 at 09:00 pm CET (08:00 pm GMT; 04:00 pm EDT) (Press release, MorphoSys, MAR 9, 2023, View Source;messagePartId=0.1 [SID1234628469]).

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MorphoSys’ Management team will host a conference call and webcast on March 16, 2023 at 01:00 pm CET (12:00 pm GMT; 8:00 am EDT) to present the results for the financial year 2022 and provide an outlook for 2023.

The conference call will start with a presentation by the Management team followed by a Q&A session.

A live webcast and slides will be made available at the Investors section on MorphoSys’ website, www.morphosys.com.

To join the conference call via phone, participants may pre-register and will receive dedicated dial-in details to easily and quickly access the call via the following website:

View Source 8&linkSecurityString=469447192