On February 16, 2023 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), an AI-driven innovator of precision antibody immunotherapies, reported the disclosure of MUC16 as the target of it’s latest immune-oncology program (Press release, iBioPharma, FEB 16, 2023, View Source [SID1234627333]).

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MUC16 is a well-known cancer target often overexpressed in several types of solid tumors, including ovarian, lung, and pancreas cancers. Specifically, MUC16 is a large extracellular protein expressed on more than 80% of ovarian tumors. Tumor cells can evade immune attack by shedding or glycosylating MUC16, making it difficult for traditional antibody therapies to effectively target and destroy the cancer cells.

Using its patented epitope steering AI platform, iBio’s innovative approach to this challenge allows its new monoclonal antibodies (mAbs) to bind to a specific region of MUC16 that is not shed or glycosylated, circumventing both tumor evasion mechanisms and potentially providing a powerful tool in the fight against cancer.

"The targeting of a very specific, patho-physiologically relevant, region of MUC16 is a testament to the versatility of our AI technology, as it successfully shows it can be applied to a broad range of targets," said iBio’s Interim Chief Executive Officer and Chief Scientific Officer, Martin Brenner. "This success adds to the growing list of target classes to which we have made differentiated antibodies using our patented epitope steering technology."

With the addition of the MUC16 program, iBio expands its Immuno-Oncology Portfolio complementing its Treg depleting programs IBIO-101 and CCR8, as well as its program targeting the tumor-specific EGFRvIII protein.

On February 16, 2023 Helocyte, Inc., ("Helocyte") a subsidiary company of Fortress Biotech, Inc. (Nasdaq: FBIO), reported that data from a Phase 1 pilot trial (see NCT03560752) evaluating the potential safety, immunological response and efficacy of the cytomegalovirus ("CMV") vaccine Triplex to enhance CMV protective immunity in immunosuppressed recipients of allogeneic hematopoietic cell transplants ("HCT") will be presented at the 2023 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCTÔ and CIBMTRâ ("Tandem Meetings"), taking place February 15-19, 2023, in Orlando, Florida (Press release, Helocyte, FEB 16, 2023, View Source [SID1234627332]). Triplex was developed by City of Hope, one of the largest cancer research and treatment organizations in the United States, and exclusively licensed to Helocyte in 2015.

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City of Hope researchers led a pilot trial to explore the vaccination of immunocompetent HCT donors with Triplex to enhance CMV protective immunity in recipients of an allogeneic HCT. Triplex is a recombinant modified vaccinia Ankara ("MVA") viral vector expressing immunodominant CMV antigens, pp65, IE1 and IE2 that has previously been demonstrated to be safe, highly immunogenic and potentially efficacious in both healthy volunteers and HCT recipients. Triplex has been dosed safely in over 100 subjects.

"CMV is still the most common infectious complication in allogeneic HCT and remains of fundamental clinical concern. While preemptive antiviral therapies have greatly reduced the risk of CMV end-organ disease and mortality, such therapies are often associated with significant toxicities, delayed immune reconstitution and even resistance," said Ryotaro Nakamura, M.D., Jan & Mace Siegel Professor in Hematology & Hematopoietic Cell Transplantation, City of Hope. "This novel approach represents a promising strategy to convey CMV protective immunity to HCT recipients early after transplant, and potentially reduce or eliminate the need for antiviral therapy."

The trial, the results of which were also recently published in the American Journal of Hematology, enrolled 17 CMV-seropositive patients who received an HCT from a CMV-seropositive (n=16) or CMV-seronegative (n=1) matched related donor ("MRD") vaccinated with Triplex prior to stem cell harvest.[i] Donor and recipient pairs who participated in the trial were observed to have adhered closely to the protocol. Triplex was generally well-tolerated in stem cell donors who participated in the study with no serious adverse events reported. All HCT recipients were fully engrafted with stem cells of donor origin without delay. On day 28 post-HCT, levels of functional CMV and vaccine-specific CD137+CD8+ T cells were observed to be significantly higher (p=0.017 and for pp65 alone, p<0.0001) in recipients of Triplex-vaccinated MRD compared to a control cohort of recipients of HCT from an unvaccinated MRD. CMV events requiring antiviral intervention in recipients with Triplex-vaccinated donors were observed to be lower (18%) than those in similar cohorts prophylactically treated with the antiviral, letermovir (37%).

Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We are encouraged by the results of this pilot study that further demonstrate the potential safety, immunogenicity, and efficacy of Triplex. The donor vaccination paradigm deployed in the trial may be applicable to other (higher risk) HCT settings, including those involving haploidentical or mis-matched donors – thereby potentially reducing the need for antivirals, which have been associated with significant toxicities and delayed immune reconstitution. We look forward to advancing the development of Triplex, which is currently the subject of multiple ongoing and planned clinical trials in HCT, solid organ transplantation, Human Immunodeficiency Virus and in combination with CAR-T therapy, most of which are supported by funding from the National Institutes of Health."

Details of the presentation are as follows:

Abstract number: 82
Title: CMV-MVA Triplex Vaccination of Stem Cell Donors to Enhance CMV Specific Immunity and Prevent CMV Viremia in Recipients after Stem Cell Transplant
Date and Time: Saturday, February 18, 11:00 a.m. – 11:15 a.m. ET
Location: World Center Marriott – Crystal NPQ
Presenter: Ryotaro Nakamura, M.D., Jan & Mace Siegel Professor in Hematology & Hematopoietic Cell Transplantation, City of Hope, Duarte, CA
For more information about the 2023 Tandem Meetings, please visit: View Source

About Triplex
Triplex is a universal (non-HLA-restricted) recombinant Modified Vaccinia Ankara viral vector vaccine engineered to induce a robust and durable virus-specific T cell response to three immuno-dominant proteins [UL83 (pp65), UL123 (IE1), UL122 (IE2)] linked to CMV complications in the post-transplant setting. In previous Phase 1 and Phase 2 studies, Triplex was found to be safe, well-tolerated and highly immunogenic. Triplex is currently the subject of multiple ongoing clinical trials, including: a Phase 2 trial for CMV control in HCT recipients with haploidentical donors (see NCT04060277); a Phase 1/2 trial for CMV control in pediatric recipients of HCT (see NCT03354728); a Phase 2 trial for reduction in viral load of Human Immunodeficiency Virus ("HIV") in adults co-infected with HIV and CMV (see NCT05099965); and a Phase 1 trial of Triplex in combination with a bi-specific CMV/CD-19 Chimeric Antigen Receptor T Cell for the treatment of Non-Hodgkin Lymphoma (see NCT05432635). Triplex is also the subject of several planned studies, including: a Phase 2 trial for CMV control in HCT recipients in which the donor is vaccinated with Triplex; a Phase 2 for CMV control in recipients of liver transplant; and a Phase 2 trial for CMV control in recipients of kidney transplant.

On February 16, 2023 Fresenius Kabi reported that its Biosimilar Stimufend (pegfilgrastim-fpgk) is now available from Fresenius Kabi in the United States (Press release, Fresenius, FEB 16, 2023, View Source [SID1234627331]). Stimufend was approved by the U.S. Food and Drug Administration (FDA) in September 2022 for use in patients with non-myeloid malignancies receiving myelosuppressive anticancer drugs associated with a clinically significant incidence of febrile neutropenia. It is Fresenius Kabi’s first U.S. biosimilar launch. The expansion of the company’s global biosimilars portfolio with a focus on oncology and immunology is an important milestone in its Vision 2026 growth strategy.

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On February 16, 2023 Zumutor reported that it received a notice of allowance from USPTO on its flagship product : ZM008 (Press release, Zumutor Biologics, FEB 16, 2023, View Source [SID1234627328]).

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ZM008 is a novel monoclonal antibody: designed and developed by scientists at Zumutor Biologics.

This Ab will have far reaching impact on patient outcomes in solid cancers especially those with limited standard therapeutic options.

Clinical trials will commence soon in the US.

On February 16, 2023 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the full year ended December 31, 2022 and provided a business update (Press release, Ascendis Pharma, FEB 16, 2023, View Source [SID1234627326]).

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"Our unique TransCon technology platform and algorithm for product innovation enables us to address major unmet medical needs with a diverse, growing pipeline of highly differentiated product candidates," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "With our demonstrated ability to take a product from concept through approval and launch, we will continue to focus on building long term value for patients and other stakeholders, establishing Ascendis Pharma as a leading sustainable, profitable biopharma company."

Select Highlights & Anticipated 2023 Milestones

TransCon hGH:
First European SKYTROFA (lonapegsomatropin) commercial launch in Germany on track for the third quarter of 2023.
In the third quarter of 2023, we anticipate completing enrollment in New InsiGHTS, a four-armed Phase 2 trial designed to investigate the safety, tolerability, and efficacy of different dose levels of TransCon hGH in patients with Turner Syndrome.
We expect Phase 3 topline results from foresiGHt in adult growth hormone deficiency in the fourth quarter of 2023.
Fourth quarter 2022 SKYTROFA (lonapegsomatropin-tcgd) U.S. revenue grew to €17.1 million.
TransCon PTH:
U.S. FDA Priority Review continues for use in adult patients with hypoparathyroidism, with a PDUFA date of April 30, 2023. If approved, U.S. commercial launch expected by the end of the second quarter of 2023.
European Commission decision on MAA anticipated during the fourth quarter of 2023. If approved, first European country launch expected in early 2024.
In anticipation of U.S. and EU approvals, commercial, medical affairs, product supply and other teams continue launch readiness activities.
Phase 3 PaTHway Japan trial achieved its primary objectives; topline results consistent with North American and EU trials.
Enrollment opened in January 2023 for U.S. Expanded Access Program.
Q1-2022 Q2-2022 Q3-2022 Q4-2022 2022
SKYTROFA revenue (millions) € 1.9 € 4.4 € 12.3 € 17.1 € 35.7

TransCon CNP:
Announced positive topline data from the Phase 2 ACcomplisH Trial, with results in children with achondroplasia down to 2 years of age; as of February 14, 2023 all 57 patients currently remain in the trial with treatment duration up to 3 years.
During the second quarter of 2023, we expect to complete enrollment in ApproaCH, a global randomized, double-blind, placebo-controlled Phase 2b trial in children ages 2–11 years with achondroplasia. The trial targets enrollment of ~80 patients.
During the third quarter of 2023, we plan to submit an IND or similar in children under the age of two years with achondroplasia.
TransCon TLR7/8 Agonist:
Reported topline data and recommended Phase 2 dose from the dose escalation portion of the Phase 1/2 transcendIT-101 Trial. Early signs of clinical activity were observed in patients receiving TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab.
Enrollment in the dose expansion phase of transcendIT-101 continues, with a focus on investigating TransCon TLR7/8 Agonist in combination with pembrolizumab in four different cancer types.
TransCon IL-2 β/γ:
The Phase 1/2 IL-βelieγe Trial evaluating TransCon IL-2 β/γ monotherapy in patients with locally advanced or metastatic solid tumors continues to enroll patients. Results from monotherapy dose escalation are expected during the first quarter of 2023. Dose escalation combination therapy results expected during the third quarter of 2023.
Preparing to initiate βelieγe-IT-201, a randomized Phase 2 trial of TransCon IL-2 β/γ and TLR7/8 combination therapies, in the second quarter of 2023.
TransCon RBZ:
Ophthalmology selected as the third therapeutic area; TransCon RBZ (ranibizumab) selected as the first investigational pipeline candidate, designed for higher efficacy with 6-month dosing intervals.
Ended the fourth quarter of 2022 with cash, cash equivalents, and marketable securities totaling €742.9 million.
Full-Year 2022 Financial Results

Total revenue for 2022 was €51.2 million compared to €7.8 million in 2021. Revenue for 2022 include SKYTROFA U.S. revenue, and license, clinical supply and services provided to third parties, primarily VISEN Pharmaceuticals. Revenue in 2022 benefited from a full-year contribution of SKYTROFA U.S. revenue of €35.7 million compared to €0.9 million in 2021.

Research and development (R&D) costs for 2022 were €379.6 million compared to €295.9 million in 2021. The higher R&D costs in 2022 reflect a one-time reversal of pre-launch inventories in 2021, following the U.S. FDA approval of SKYTROFA in August 2021. In addition, higher R&D costs in 2022 reflect manufacturing of pre-launch inventories for TransCon PTH and an increase in employee and other costs attributable to organizational growth.

Selling, general, and administrative (SG&A) expenses for 2022 were €221.2 million compared to €160.2 million in 2021. Higher SG&A expenses were primarily due to an increase in commercial and administrative personnel following the launch of SKYTROFA in the U.S. and preparation for future product launches.

Our share of net loss of associate was €17.7 million in 2022, compared to a net gain of €12.0 million in 2021. For 2021, the net profit of associate included a non-cash gain of €42.3 million as a result of a financing round in VISEN.

Net finance income was €1.7 million in 2022 compared to a net finance income of €55.8 million in 2021.

For the full year 2022, Ascendis Pharma reported a net loss of €583.2 million, or €10.40 per share (basic and diluted) compared to a net loss of €383.6 million, or €7.00 per share (basic and diluted) for the same period in 2021.

As of December 31, 2022, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €742.9 million compared to €789.6 million as of December 31, 2021. As of December 31, 2022, Ascendis Pharma had 57,152,295 ordinary shares outstanding.

Conference Call and Webcast Information

Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its full year 2022 financial results.

Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at View Source A replay of the webcast will be available on this section of our website shortly after conclusion of the event for 30 days.